| scholarly article | Q13442814 |
| review article | Q7318358 |
| P2093 | author name string | Katherine A High | |
| Federico Mingozzi | |||
| P2860 | cites work | Novel adeno-associated viruses from rhesus monkeys as vectors for human gene therapy | Q24535690 |
| Vision 1 year after gene therapy for Leber's congenital amaurosis | Q24607981 | ||
| Treatment of leber congenital amaurosis due to RPE65 mutations by ocular subretinal injection of adeno-associated virus gene vector: short-term results of a phase I trial | Q24608049 | ||
| Transfusion independence and HMGA2 activation after gene therapy of human β-thalassaemia | Q24618997 | ||
| Gene therapy for Leber's congenital amaurosis is safe and effective through 1.5 years after vector administration | Q24627838 | ||
| Safety and efficacy of gene transfer for Leber's congenital amaurosis | Q24634724 | ||
| LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1 | Q28210584 | ||
| Fatal systemic inflammatory response syndrome in a ornithine transcarbamylase deficient patient following adenoviral gene transfer | Q28211178 | ||
| The danger model: a renewed sense of self | Q28214633 | ||
| Current prospects for RNA interference-based therapies | Q28235733 | ||
| Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a phase 1 dose-escalation trial | Q28262431 | ||
| Effect of gene therapy on visual function in Leber's congenital amaurosis | Q28277981 | ||
| Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1 | Q28290118 | ||
| Genome editing with engineered zinc finger nucleases | Q28290795 | ||
| Gene delivery of AAV2-neurturin for Parkinson's disease: a double-blind, randomised, controlled trial | Q28296434 | ||
| AAV2-GAD gene therapy for advanced Parkinson's disease: a double-blind, sham-surgery controlled, randomised trial | Q28307713 | ||
| Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response | Q29619095 | ||
| Liver-directed recombinant adeno-associated viral gene delivery rescues a lethal mouse model of methylmalonic acidemia and provides long-term phenotypic correction | Q30496514 | ||
| Gene therapy with novel adeno-associated virus vectors substantially diminishes atherosclerosis in a murine model of familial hypercholesterolemia | Q30935623 | ||
| Investigation of the cause of death in a gene-therapy trial | Q33385315 | ||
| Retrovirus gene therapy for X-linked chronic granulomatous disease can achieve stable long-term correction of oxidase activity in peripheral blood neutrophils | Q33387329 | ||
| Molecular signatures of disease brain endothelia provide new sites for CNS-directed enzyme therapy | Q33502598 | ||
| Directed evolution of a novel adeno-associated virus (AAV) vector that crosses the seizure-compromised blood-brain barrier (BBB). | Q33521174 | ||
| Review of the clinical development of alipogene tiparvovec gene therapy for lipoprotein lipase deficiency | Q33567468 | ||
| Several log increase in therapeutic transgene delivery by distinct adeno-associated viral serotype vectors | Q45873434 | ||
| Investigation of chronic hepatitis C infection in individuals with haemophilia: assessment of invasive and non-invasive methods | Q45877891 | ||
| A deletion mutation causes hemophilia B in Lhasa Apso dogs | Q45879429 | ||
| Lack of germline transmission of vector sequences following systemic administration of recombinant AAV-2 vector in males | Q45881920 | ||
| Worldwide epidemiology of neutralizing antibodies to adeno-associated viruses. | Q45882349 | ||
| Gene therapy for immunodeficiency due to adenosine deaminase deficiency. | Q45882856 | ||
| A coagulation factor IX-deficient mouse model for human hemophilia B. | Q45884173 | ||
| Sustained phenotypic correction of hemophilia B dogs with a factor IX null mutation by liver-directed gene therapy | Q45884700 | ||
| Uniform scale-independent gene transfer to striated muscle after transvenular extravasation of vector | Q45886806 | ||
| Influence of vector dose on factor IX-specific T and B cell responses in muscle-directed gene therapy | Q45888356 | ||
| CSF biomarkers and incipient Alzheimer disease in patients with mild cognitive impairment. | Q45923691 | ||
| Risk factors for hemorrhage during microelectrode-guided deep brain stimulator implantation for movement disorders | Q46518059 | ||
| Aspartoacylase deficiency and N-acetylaspartic aciduria in patients with canavan disease | Q48115232 | ||
| Immune responses to AAV in a phase I study for Canavan disease | Q48620414 | ||
| A phase II, double-blind, randomized, placebo-controlled clinical trial of tgAAVCF using maxillary sinus delivery in patients with cystic fibrosis with antrostomies. | Q51708365 | ||
| Repeated Aerosolized AAV-CFTR for Treatment of Cystic Fibrosis: A Randomized Placebo-Controlled Phase 2B Trial | Q57952103 | ||
| Gene therapy and the germline | Q74623212 | ||
| Efficient and persistent gene transfer of AAV-CFTR in maxillary sinus | Q77292224 | ||
| Case 15-2009: a man with coma after cardiac arrest | Q95802924 | ||
| Efficient, long-term hepatic gene transfer using clinically relevant HDAd doses by balloon occlusion catheter delivery in nonhuman primates | Q33713289 | ||
| Host and vector-dependent effects on the risk of germline transmission of AAV vectors | Q33713716 | ||
| Intravenous administration of self-complementary AAV9 enables transgene delivery to adult motor neurons | Q33713769 | ||
| Persistent expression of FLAG-tagged micro dystrophin in nonhuman primates following intramuscular and vascular delivery | Q33730464 | ||
| Safety and tolerability of putaminal AADC gene therapy for Parkinson disease | Q33731687 | ||
| Absence of a desmopressin response after therapeutic expression of factor VIII in hemophilia A dogs with liver-directed neonatal gene therapy | Q33770970 | ||
| Identifying photoreceptors in blind eyes caused by RPE65 mutations: Prerequisite for human gene therapy success | Q33771032 | ||
| Hepatocyte-targeted HFE and TFR2 control hepcidin expression in mice | Q33806074 | ||
| Peripheral transvenular delivery of adeno-associated viral vectors to skeletal muscle as a novel therapy for hemophilia B. | Q33931862 | ||
| RNA interference improves motor and neuropathological abnormalities in a Huntington's disease mouse model | Q33936969 | ||
| Safety of AAV factor IX peripheral transvenular gene delivery to muscle in hemophilia B dogs. | Q34025098 | ||
| Functional correction of established central nervous system deficits in an animal model of lysosomal storage disease with feline immunodeficiency virus-based vectors | Q34028579 | ||
| Clinical protocol. Gene therapy of Canavan disease: AAV-2 vector for neurosurgical delivery of aspartoacylase gene (ASPA) to the human brain | Q34142832 | ||
| AAV-mediated factor IX gene transfer to skeletal muscle in patients with severe hemophilia B. | Q34168942 | ||
| Characterization of a recombinant adeno-associated virus type 2 Reference Standard Material | Q34211507 | ||
| Sustained alpha-sarcoglycan gene expression after gene transfer in limb-girdle muscular dystrophy, type 2D. | Q34266296 | ||
| Canine hemophilia B resulting from a point mutation with unusual consequences | Q34326780 | ||
| Adeno-associated virus vectors can be efficiently produced without helper virus | Q34479933 | ||
| Adeno-associated viral vector-mediated gene transfer results in long-term enzymatic and functional correction in multiple organs of Fabry mice | Q34483749 | ||
| Fatality in mice due to oversaturation of cellular microRNA/short hairpin RNA pathways | Q34530552 | ||
| Safety and tolerability of gene therapy with an adeno-associated virus (AAV) borne GAD gene for Parkinson's disease: an open label, phase I trial | Q34640765 | ||
| Results from a phase I safety trial of hAADC gene therapy for Parkinson disease | Q34656075 | ||
| Prophylaxis versus episodic treatment to prevent joint disease in boys with severe hemophilia | Q34661163 | ||
| Assessing the potential for AAV vector genotoxicity in a murine model | Q34754355 | ||
| Adeno-Associated Virus Serotype 8 Gene Transfer Rescues a Neonatal Lethal Murine Model of Propionic Acidemia | Q34774811 | ||
| Treatment of late infantile neuronal ceroid lipofuscinosis by CNS administration of a serotype 2 adeno-associated virus expressing CLN2 cDNA. | Q34778098 | ||
| Leber congenital amaurosis: genes, proteins and disease mechanisms | Q34796759 | ||
| Induction of immune tolerance to coagulation factor IX antigen by in vivo hepatic gene transfer | Q34985105 | ||
| Hematopoietic stem cell gene therapy with a lentiviral vector in X-linked adrenoleukodystrophy | Q35011497 | ||
| Robust systemic transduction with AAV9 vectors in mice: efficient global cardiac gene transfer superior to that of AAV8. | Q35025591 | ||
| Current status of gene therapy for inherited lung diseases | Q35043830 | ||
| Convection-enhanced delivery of macromolecules in the brain | Q35091232 | ||
| Recombinant adeno-associated virus type 2, 4, and 5 vectors: transduction of variant cell types and regions in the mammalian central nervous system. | Q35111738 | ||
| Leber hereditary optic neuropathy gene therapy clinical trial recruitment: year 1. | Q35561447 | ||
| Safe and efficient transduction of the liver after peripheral vein infusion of self-complementary AAV vector results in stable therapeutic expression of human FIX in nonhuman primates | Q35628868 | ||
| Safety and biological efficacy of an adeno-associated virus vector-cystic fibrosis transmembrane regulator (AAV-CFTR) in the cystic fibrosis maxillary sinus. | Q41746617 | ||
| Effects of transient immunosuppression on adenoassociated, virus-mediated, liver-directed gene transfer in rhesus macaques and implications for human gene therapy | Q41999615 | ||
| Correction of neurological disease of mucopolysaccharidosis IIIB in adult mice by rAAV9 trans-blood-brain barrier gene delivery | Q42059455 | ||
| Cross-presentation of adeno-associated virus serotype 2 capsids activates cytotoxic T cells but does not render hepatocytes effective cytolytic targets | Q42613058 | ||
| Hemophilia gene therapy: a Holy Grail found | Q42649096 | ||
| Safety and tolerability of intraputaminal delivery of CERE-120 (adeno-associated virus serotype 2-neurturin) to patients with idiopathic Parkinson's disease: an open-label, phase I trial | Q42650874 | ||
| Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes | Q42739394 | ||
| Therapeutic levels of functional human factor X in rats after retroviral-mediated hepatic gene therapy. | Q42803722 | ||
| Safety, tolerability, and clinical outcomes after intraarticular injection of a recombinant adeno-associated vector containing a tumor necrosis factor antagonist gene: results of a phase 1/2 Study | Q43209177 | ||
| Adeno-associated virus-mediated rhodopsin replacement provides therapeutic benefit in mice with a targeted disruption of the rhodopsin gene. | Q43262568 | ||
| Limb-girdle muscular dystrophy type 2D gene therapy restores alpha-sarcoglycan and associated proteins | Q43268180 | ||
| Intramuscular administration of AAV1-lipoprotein lipase S447X lowers triglycerides in lipoprotein lipase-deficient patients | Q43466855 | ||
| Gene therapy restores vision in a canine model of childhood blindness | Q43590733 | ||
| Local delivery of a recombinant adenoassociated vector containing a tumour necrosis factor alpha antagonist gene in inflammatory arthritis: a phase 1 dose-escalation safety and tolerability study. | Q43632894 | ||
| A phase I study of aerosolized administration of tgAAVCF to cystic fibrosis subjects with mild lung disease | Q43670160 | ||
| Targeting lentiviral vector expression to hepatocytes limits transgene-specific immune response and establishes long-term expression of human antihemophilic factor IX in mice | Q43702736 | ||
| Prevention of systemic clinical disease in MPS VII mice following AAV-mediated neonatal gene transfer | Q43747170 | ||
| Sustained correction of disease in naive and AAV2-pretreated hemophilia B dogs: AAV2/8-mediated, liver-directed gene therapy | Q43801966 | ||
| Timing of therapeutic intervention determines functional and survival outcomes in a mouse model of late infantile batten disease | Q44039833 | ||
| Inadvertent germline transmission of AAV2 vector: findings in a rabbit model correlate with those in a human clinical trial | Q44328165 | ||
| Immediate and long-term safety of recombinant adeno-associated virus injection into the nonhuman primate muscle | Q44474527 | ||
| Gene therapy for choroideremia: in vitro rescue mediated by recombinant adenovirus | Q44752242 | ||
| AAV2 vector harboring a liver-restricted promoter facilitates sustained expression of therapeutic levels of alpha-galactosidase A and the induction of immune tolerance in Fabry mice. | Q44753961 | ||
| Evasion of immune responses to introduced human acid alpha-glucosidase by liver-restricted expression in glycogen storage disease type II. | Q45259698 | ||
| CERE-110, an adeno-associated virus-based gene delivery vector expressing human nerve growth factor for the treatment of Alzheimer's disease. | Q45376997 | ||
| CD8(+) T-cell responses to adeno-associated virus capsid in humans | Q45406111 | ||
| Enhanced survival of the LINCL mouse following CLN2 gene transfer using the rh.10 rhesus macaque-derived adeno-associated virus vector. | Q45408947 | ||
| Phase I trial of intramuscular injection of a recombinant adeno-associated virus serotype 2 alphal-antitrypsin (AAT) vector in AAT-deficient adults | Q45409947 | ||
| Heparin binding directs activation of T cells against adeno-associated virus serotype 2 capsid | Q45414481 | ||
| Correction of feline lipoprotein lipase deficiency with adeno-associated virus serotype 1-mediated gene transfer of the lipoprotein lipase S447X beneficial mutation | Q45416450 | ||
| Early, sustained efficacy of adeno-associated virus vector-mediated gene therapy in glycogen storage disease type Ia. | Q45417049 | ||
| Adeno-associated virus vector serotypes mediate sustained correction of bilirubin UDP glucuronosyltransferase deficiency in rats | Q45418234 | ||
| Therapeutic level of functional human alpha 1 antitrypsin (hAAT) secreted from murine muscle transduced by adeno-associated virus (rAAV1) vector | Q45419136 | ||
| Transduction characteristics of adeno-associated virus vectors expressing cap serotypes 7, 8, 9, and Rh10 in the mouse brain | Q45420843 | ||
| Functional correction of CNS phenotypes in a lysosomal storage disease model using adeno-associated virus type 4 vectors. | Q45423330 | ||
| Efficient hepatic delivery and expression from a recombinant adeno-associated virus 8 pseudotyped alpha1-antitrypsin vector | Q45428790 | ||
| Adeno-associated virus terminal repeat (TR) mutant generates self-complementary vectors to overcome the rate-limiting step to transduction in vivo | Q45709105 | ||
| Chromosomal effects of adeno-associated virus vector integration | Q45734034 | ||
| Quantitative analysis of the packaging capacity of recombinant adeno-associated virus | Q45766162 | ||
| Long-term correction of canine hemophilia B by gene transfer of blood coagulation factor IX mediated by adeno-associated viral vector | Q45855552 | ||
| Intrathecal administration of AAV vectors for the treatment of lysosomal storage in the brains of MPS I mice | Q45856196 | ||
| Correction of Hunter syndrome in the MPSII mouse model by AAV2/8-mediated gene delivery | Q45856355 | ||
| AAV8-mediated expression of glucocerebrosidase ameliorates the storage pathology in the visceral organs of a mouse model of Gaucher disease. | Q45856667 | ||
| Long-term correction of ammonia metabolism and prolonged survival in ornithine transcarbamylase-deficient mice following liver-directed treatment with adeno-associated viral vectors. | Q45858049 | ||
| Reversal of type 1 diabetes by engineering a glucose sensor in skeletal muscle. | Q45858699 | ||
| Evidence of multiyear factor IX expression by AAV-mediated gene transfer to skeletal muscle in an individual with severe hemophilia B. | Q45859498 | ||
| Sustained phenotypic correction of canine hemophilia A using an adeno-associated viral vector | Q45862203 | ||
| Immune response after neonatal transfer of a human factor IX-expressing retroviral vector in dogs, cats, and mice | Q45863749 | ||
| One of three successfully treated CGD patients in a Swiss-German gene therapy trial died due to his underlying disease: A position statement from the European Society of Gene Therapy (ESGT). | Q45864119 | ||
| Pre-existing AAV capsid-specific CD8+ T cells are unable to eliminate AAV-transduced hepatocytes | Q45865401 | ||
| Targeted disruption of the mouse factor VIII gene produces a model of haemophilia A | Q45868121 | ||
| AAV vector integration sites in mouse hepatocellular carcinoma. | Q45869780 | ||
| Clinical gene transfer studies for hemophilia A. | Q35760801 | ||
| Self-complementary adeno-associated virus vectors containing a novel liver-specific human factor IX expression cassette enable highly efficient transduction of murine and nonhuman primate liver | Q35848563 | ||
| Adeno-associated virus type 2 (AAV2) capsid-specific cytotoxic T lymphocytes eliminate only vector-transduced cells coexpressing the AAV2 capsid in vivo | Q35914452 | ||
| Comprehensive care for haemophilia around the world | Q35915629 | ||
| Modulation of tolerance to the transgene product in a nonhuman primate model of AAV-mediated gene transfer to liver. | Q36007714 | ||
| Safety and efficacy of factor IX gene transfer to skeletal muscle in murine and canine hemophilia B models by adeno-associated viral vector serotype 1. | Q36438711 | ||
| Rapid uncoating of vector genomes is the key to efficient liver transduction with pseudotyped adeno-associated virus vectors | Q36509889 | ||
| A factor IX-deficient mouse model for hemophilia B gene therapy | Q36602472 | ||
| Long-term restoration of rod and cone vision by single dose rAAV-mediated gene transfer to the retina in a canine model of childhood blindness | Q36825024 | ||
| Expression of human factor IX in mice after injection of genetically modified myoblasts | Q36950396 | ||
| Silencing of T lymphocytes by antigen-driven programmed death in recombinant adeno-associated virus vector-mediated gene therapy | Q37066228 | ||
| Efficient gene therapy-based method for the delivery of therapeutics to primate cortex | Q37114888 | ||
| Manufacturing and characterizing AAV-based vectors for use in clinical studies | Q37140657 | ||
| Capsid antigen presentation flags human hepatocytes for destruction after transduction by adeno-associated viral vectors | Q37209197 | ||
| Correction of murine PKU following AAV-mediated intramuscular expression of a complete phenylalanine hydroxylating system | Q37221688 | ||
| Cellular immune response to cryptic epitopes during therapeutic gene transfer | Q37250034 | ||
| AAV-1-mediated gene transfer to skeletal muscle in humans results in dose-dependent activation of capsid-specific T cells | Q37348251 | ||
| Sustained transgene expression despite T lymphocyte responses in a clinical trial of rAAV1-AAT gene therapy | Q37364038 | ||
| Calcium upregulation by percutaneous administration of gene therapy in cardiac disease (CUPID Trial), a first-in-human phase 1/2 clinical trial. | Q37364786 | ||
| Successful expression of human factor IX following repeat administration of adenoviral vector in mice. | Q37685172 | ||
| Newborn screening for treatable genetic conditions: past, present and future | Q37759830 | ||
| Manufacturing and regulatory strategies for clinical AAV2-hRPE65. | Q37780248 | ||
| Ex vivo gene transfer and correction for cell-based therapies | Q37858878 | ||
| State-of-the-art gene-based therapies: the road ahead | Q37862074 | ||
| Phase 1 trial of FVIII gene transfer for severe hemophilia A using a retroviral construct administered by peripheral intravenous infusion | Q38520320 | ||
| Undetectable transcription of cap in a clinical AAV vector: implications for preformed capsid in immune responses | Q39501488 | ||
| Proteasome inhibitors decrease AAV2 capsid derived peptide epitope presentation on MHC class I following transduction. | Q39638928 | ||
| Lack of immunotoxicity after regional intravenous (RI) delivery of rAAV to nonhuman primate skeletal muscle | Q39639059 | ||
| Ubiquitination of both adeno-associated virus type 2 and 5 capsid proteins affects the transduction efficiency of recombinant vectors. | Q39682659 | ||
| Nucleotide sequence and organization of the adeno-associated virus 2 genome | Q40143539 | ||
| Recombinant adeno-associated virus mediated gene transfer in a mouse model for homocystinuria | Q40187095 | ||
| The burden of genetic disease on inpatient care in a children's hospital | Q40542360 | ||
| AAV serotype 2 vectors preferentially integrate into active genes in mice | Q40643545 | ||
| Efficient transfection of primary cells in a canine hemophilia B model using adenovirus-polylysine-DNA complexes | Q41484655 | ||
| Haemophilia prophylaxis in young patients--a long-term follow-up | Q41497194 | ||
| Dystrophin immunity in Duchenne's muscular dystrophy. | Q41590387 | ||
| Evidence for gene transfer and expression of factor IX in haemophilia B patients treated with an AAV vector. | Q41722852 | ||
| P433 | issue | 5 | |
| P921 | main subject | genetic disease | Q200779 |
| P304 | page(s) | 341-355 | |
| P577 | publication date | 2011-05-01 | |
| P1433 | published in | Nature Reviews Genetics | Q1071824 |
| P1476 | title | Therapeutic in vivo gene transfer for genetic disease using AAV: progress and challenges | |
| P478 | volume | 12 |
| Q89733487 | 10th European immunogenicity platform open symposium on immunogenicity of biopharmaceuticals |
| Q41171772 | A Five-Repeat Micro-Dystrophin Gene Ameliorated Dystrophic Phenotype in the Severe DBA/2J-mdx Model of Duchenne Muscular Dystrophy |
| Q35837765 | A Novel Adeno-Associated Virus-Based Genetic Vaccine Encoding the Hepatitis C Virus NS3/4 Protein Exhibits Immunogenic Properties in Mice Superior to Those of an NS3-Protein-Based Vaccine |
| Q55508466 | A Recombinant Baculovirus Efficiently Generates Recombinant Adeno-Associated Virus Vectors in Cultured Insect Cells and Larvae. |
| Q36736426 | A Regulatory Element Near the 3' End of the Adeno-Associated Virus rep Gene Inhibits Adenovirus Replication in cis by Means of p40 Promoter-Associated Short Transcripts |
| Q38719141 | A Targeted and Stable Polymeric Nanoformulation Enhances Systemic Delivery of mRNA to Tumors |
| Q33586945 | A capillary electrophoresis sequencing method for the identification of mutations in the inverted terminal repeats of adeno-associated virus |
| Q38972131 | A chemical switch for controlling viral infectivity |
| Q42322592 | A comparative study on nonviral genetic modifications in cord blood and bone marrow mesenchymal stem cells. |
| Q36870087 | A highly secreted sulphamidase engineered to cross the blood-brain barrier corrects brain lesions of mice with mucopolysaccharidoses type IIIA. |
| Q39401822 | A largely random AAV integration profile after LPLD gene therapy |
| Q38833030 | A magic bullet to specifically eliminate mutated mitochondrial genomes from patients' cells |
| Q24599954 | A new generation of human artificial chromosomes for functional genomics and gene therapy |
| Q35751578 | A simplified immune suppression scheme leads to persistent micro-dystrophin expression in Duchenne muscular dystrophy dogs |
| Q37313599 | A translationally optimized AAV-UGT1A1 vector drives safe and long-lasting correction of Crigler-Najjar syndrome |
| Q28468539 | A universal system to select gene-modified hepatocytes in vivo |
| Q40543766 | AAV Delivery of Endothelin-1 shRNA Attenuates Cold-Induced Hypertension |
| Q40173850 | AAV Gene Therapy for Alcoholism: Inhibition of Mitochondrial Aldehyde Dehydrogenase Enzyme Expression in Hepatoma Cells |
| Q92876148 | AAV Vector Immunogenicity in Humans: A Long Journey to Successful Gene Transfer |
| Q37498000 | AAV Vectors Vaccines Against Infectious Diseases. |
| Q36298638 | AAV vectors containing rDNA homology display increased chromosomal integration and transgene persistence. |
| Q38828004 | AAV-ID: A Rapid and Robust Assay for Batch-to-Batch Consistency Evaluation of AAV Preparations |
| Q92863325 | AAV-Mediated CRISPR/Cas9 Gene Editing in Murine Phenylketonuria |
| Q92666846 | AAV-PHP.B Administration Results in a Differential Pattern of CNS Biodistribution in Non-human Primates Compared with Mice |
| Q40052760 | AAV-PHP.B-Mediated Global-Scale Expression in the Mouse Nervous System Enables GBA1 Gene Therapy for Wide Protection from Synucleinopathy |
| Q36228880 | AAV-based RNAi silencing of NADPH oxidase gp91(phox) attenuates cold-induced cardiovascular dysfunction |
| Q39137705 | AAV-mediated Sirt1 overexpression in skeletal muscle activates oxidative capacity but does not prevent insulin resistance |
| Q34214323 | AAV-mediated human PEDF inhibits tumor growth and metastasis in murine colorectal peritoneal carcinomatosis model |
| Q33836234 | AAV-mediated pancreatic overexpression of Igf1 counteracts progression to autoimmune diabetes in mice. |
| Q91652426 | AAV6 Vexosomes Mediate Robust Suicide Gene Delivery in a Murine Model of Hepatocellular Carcinoma |
| Q35568525 | AAV6-mediated systemic shRNA delivery reverses disease in a mouse model of facioscapulohumeral muscular dystrophy |
| Q96816603 | AAV8 Ins1-Cre can produce efficient β-cell recombination but requires consideration of off-target effects |
| Q42085309 | AAV8-mediated Sirt1 gene transfer to the liver prevents high carbohydrate diet-induced nonalcoholic fatty liver disease. |
| Q36588788 | AAV9-mediated central nervous system-targeted gene delivery via cisterna magna route in mice |
| Q59354464 | Accurate Titration of Infectious AAV Particles Requires Measurement of Biologically Active Vector Genomes and Suitable Controls |
| Q42150059 | Adeno-Associated Viral Vectors at the Frontier between Tolerance and Immunity |
| Q39409371 | Adeno-Associated Virus (AAV) as a Vector for Gene Therapy. |
| Q38685537 | Adeno-Associated Virus Vectors and Stem Cells: Friends or Foes? |
| Q64071676 | Adeno-Associated Virus as an Effective Malaria Booster Vaccine Following Adenovirus Priming |
| Q38856794 | Adeno-Associated Virus-Based Gene Therapy for CNS Diseases. |
| Q37593208 | Adeno-associated Virus (AAV) Assembly-Activating Protein Is Not an Essential Requirement for Capsid Assembly of AAV Serotypes 4, 5, and 11. |
| Q34370111 | Adeno-associated virus (AAV) vectors in gene therapy: immune challenges and strategies to circumvent them. |
| Q54944159 | Adeno-associated virus 2/9 delivery of Cre recombinase in mouse primary afferents. |
| Q27012320 | Adeno-associated virus at 50: a golden anniversary of discovery, research, and gene therapy success--a personal perspective |
| Q38188560 | Adeno-associated virus structural biology as a tool in vector development |
| Q27005597 | Adeno-associated virus-mediated microRNA delivery and therapeutics |
| Q38919524 | Adenoassociated virus serotype 9-mediated gene therapy for x-linked adrenoleukodystrophy |
| Q44063015 | Adenovirus-mediated overexpression of Tcfe3 ameliorates hyperglycaemia in a mouse model of diabetes by upregulating glucokinase in the liver |
| Q34472188 | Adipose tissue insulin receptor knockdown via a new primate-derived hybrid recombinant AAV serotype. |
| Q38134331 | Advances and challenges in gene-based approaches for osteoarthritis |
| Q57170592 | Advances in gene therapy for hemophilia: basis, current status, and future perspectives |
| Q38706445 | Advances in the delivery of RNA therapeutics: from concept to clinical reality. |
| Q26782706 | Advances in therapeutic CRISPR/Cas9 genome editing |
| Q30409171 | Adverse health consequences of performance-enhancing drugs: an Endocrine Society scientific statement |
| Q24562178 | Allele-specific silencing of mutant Myh6 transcripts in mice suppresses hypertrophic cardiomyopathy |
| Q27324212 | Allele-specific silencing of mutant huntingtin in rodent brain and human stem cells |
| Q37272838 | An emerging adeno-associated viral vector pipeline for cardiac gene therapy |
| Q42541172 | An engineered tale-transcription factor rescues transcription of factor VII impaired by promoter mutations and enhances its endogenous expression in hepatocytes |
| Q92995646 | Analysis of recombinant adeno-associated viral vector shedding in sheep following intracoronary delivery |
| Q27012571 | Animal models of hemophilia |
| Q83354105 | Annual banned-substance review: analytical approaches in human sports drug testing |
| Q39108774 | Antibody gene transfer with adeno-associated viral vectors as a method for HIV prevention |
| Q59339950 | Antibody-mediated prevention and treatment of HIV-1 infection |
| Q91950307 | Antibody-targeted chromatin enables effective intracellular delivery and functionality of CRISPR/Cas9 expression plasmids |
| Q57022663 | Antigen-selective modulation of AAV immunogenicity with tolerogenic rapamycin nanoparticles enables successful vector re-administration |
| Q40404779 | Antitumor effect of an adeno-associated virus expressing apolipoprotein A-1 fused to interferon alpha in an interferon alpha-resistant murine tumor model |
| Q38645275 | Application of polyploid adeno-associated virus vectors for transduction enhancement and neutralizing antibody evasion |
| Q89221788 | Applications of CRISPR-Cas Enzymes in Cancer Therapeutics and Detection |
| Q91866605 | Applications of Genome Editing Technology in Animal Disease Modeling and Gene Therapy |
| Q28703567 | Applying gene silencing technology to contraception |
| Q92654317 | Applying switchable Cas9 variants to in vivo gene editing for therapeutic applications |
| Q38826202 | Beyond the brain: Optogenetic control in the spinal cord and peripheral nervous system |
| Q41140755 | Biodegradable silicon nanoneedles delivering nucleic acids intracellularly induce localized in vivo neovascularization. |
| Q37066323 | Bioengineering of AAV2 capsid at specific serine, threonine, or lysine residues improves its transduction efficiency in vitro and in vivo. |
| Q41443741 | Biophysical and ultrastructural characterization of adeno-associated virus capsid uncoating and genome release |
| Q37277305 | CFTR gene transfer with AAV improves early cystic fibrosis pig phenotypes |
| Q39324185 | CRISPR/Cas9-mediated correction of human genetic disease. |
| Q39294371 | CRISPR/Cas9: at the cutting edge of hepatology. |
| Q92825245 | Capsid-specific removal of circulating antibodies to adeno-associated virus vectors |
| Q35174923 | Cardiac gene transfer of short hairpin RNA directed against phospholamban effectively knocks down gene expression but causes cellular toxicity in canines |
| Q41732280 | Cell type-specific delivery of short interfering RNAs by dye-functionalised theranostic nanoparticles |
| Q33938435 | Cell-Mediated Immunity to AAV Vectors, Evolving Concepts and Potential Solutions |
| Q38626721 | Challenges in carrier-mediated intracellular delivery: moving beyond endosomal barriers. |
| Q52587859 | Characterization of AAV vector particle stability at the single-capsid level. |
| Q28730955 | Characterization of a FGF19 variant with altered receptor specificity revealed a central role for FGFR1c in the regulation of glucose metabolism |
| Q24620532 | Clarifying lysosomal storage diseases |
| Q89073798 | Clinical Evaluations of Toxicity and Efficacy of Nanoparticle-Mediated Gene Therapy |
| Q37980864 | Cocaine hydrolase gene therapy for cocaine abuse |
| Q49608243 | Combination Therapy Is the New Gene Therapy? |
| Q30557598 | Comparative analysis of adeno-associated virus capsid stability and dynamics |
| Q39044467 | Comparison of Serum rAAV Serotype-Specific Antibodies in Patients with Duchenne Muscular Dystrophy, Becker Muscular Dystrophy, Inclusion Body Myositis, or GNE Myopathy |
| Q52313908 | Complement-Mediated Activation of the NLRP3 Inflammasome and Its Inhibition by AAV-Mediated Delivery of CD59 in a Model of Uveitis. |
| Q39675326 | Computationally designed liver-specific transcriptional modules and hyperactive factor IX improve hepatic gene therapy |
| Q33598920 | Construction and expression of a lentivirus expression vector carrying the VEGF165-EGFP fusion gene in breast cancer MCF-7 cells |
| Q36614445 | Creation of a cardiotropic adeno-associated virus: the story of viral directed evolution |
| Q28235733 | Current prospects for RNA interference-based therapies |
| Q38938827 | Cytotoxic genes from traditional Chinese medicine inhibit tumor growth both in vitro and in vivo |
| Q40351302 | Defining the stoichiometry and cargo load of viral and bacterial nanoparticles by Orbitrap mass spectrometry |
| Q33905659 | Deletion of the B-B' and C-C' regions of inverted terminal repeats reduces rAAV productivity but increases transgene expression |
| Q33625875 | Delivery is key: lessons learnt from developing splice-switching antisense therapies |
| Q38003108 | Detection of EPO gene doping in blood |
| Q37034685 | Determination of Anti-Adeno-Associated Viral Vector Neutralizing Antibodies in Patients With Heart Failure in the Cardiovascular Foundation of Colombia (ANVIAS): Study Protocol |
| Q39784405 | Determination of anti-adeno-associated virus vector neutralizing antibody titer with an in vitro reporter system |
| Q90361148 | Development of Versatile and Flexible Sf9 Packaging Cell Line-Dependent OneBac System for Large-Scale Recombinant Adeno-Associated Virus Production |
| Q38951348 | Differential contribution of adeno-associated virus type 2 Rep protein expression and nucleic acid elements to inhibition of adenoviral replication in cis and in trans |
| Q35194954 | Differential effects of two MRI contrast agents on the integrity and distribution of rAAV2 and rAAV5 in the rat striatum. |
| Q36270357 | Differential myofiber-type transduction preference of adeno-associated virus serotypes 6 and 9. |
| Q36740271 | Differential type I interferon-dependent transgene silencing of helper-dependent adenoviral vs. adeno-associated viral vectors in vivo |
| Q42541148 | Displaying high-affinity ligands on adeno-associated viral vectors enables tumor cell-specific and safe gene transfer |
| Q90782110 | Distinct transduction of muscle tissue in mice after systemic delivery of AAVpo1 vectors |
| Q64253691 | Dual AAV-mediated gene therapy restores hearing in a DFNB9 mouse model |
| Q42175045 | Duchenne muscular dystrophy gene therapy in the canine model |
| Q35641673 | Duchenne muscular dystrophy gene therapy in the canine model. |
| Q37685861 | Effect of bortezomib on the efficacy of AAV9.SERCA2a treatment to preserve cardiac function in a rat pressure-overload model of heart failure. |
| Q36780063 | Effects of immunosuppression on circulating adeno-associated virus capsid-specific T cells in humans |
| Q40363171 | Efficient Gene Delivery and Expression in Pancreas and Pancreatic Tumors by Capsid-Optimized AAV8 Vectors. |
| Q36967723 | Emerging therapies for mitochondrial disorders |
| Q37978779 | Emerging uses for pediatric hematopoietic stem cells |
| Q45357434 | Endocytic processing of adeno-associated virus type 8 vectors for transduction of target cells |
| Q36709834 | Engineered AAV vector minimizes in vivo targeting of transduced hepatocytes by capsid-specific CD8+ T cells. |
| Q45865170 | Engineered hair follicle mesenchymal stem cells overexpressing controlled-release insulin reverse hyperglycemia in mice with type L diabetes |
| Q52646448 | Engineering T7 bacteriophage as a potential DNA vaccine targeting delivery vector. |
| Q37213826 | Engraftment of a galactose receptor footprint onto adeno-associated viral capsids improves transduction efficiency |
| Q36951529 | Enhanced T cell function in a mouse model of human glycosylation. |
| Q89777435 | Enhanced liver gene transfer and evasion of preexisting humoral immunity with exosome-enveloped AAV vectors |
| Q34991117 | Enhanced transgene expression from recombinant single-stranded D-sequence-substituted adeno-associated virus vectors in human cell lines in vitro and in murine hepatocytes in vivo. |
| Q30401360 | Enhancement of Blood-Brain Barrier Permeability and Delivery of Antisense Oligonucleotides or Plasmid DNA to the Brain by the Combination of Bubble Liposomes and High-Intensity Focused Ultrasound |
| Q36792325 | Enhancing Transgene Expression from Recombinant AAV8 Vectors in Different Tissues Using Woodchuck Hepatitis Virus Post-Transcriptional Regulatory Element |
| Q91587743 | Enhancing the Therapeutic Potential of Sulfamidase for the Treatment of Mucopolysaccharidosis IIIA |
| Q35964228 | Enzyme replacement in the CSF to treat metachromatic leukodystrophy in mouse model using single intracerebroventricular injection of self-complementary AAV1 vector |
| Q37858878 | Ex vivo gene transfer and correction for cell-based therapies |
| Q52559115 | Exon Specific U1 snRNAs improve ELP1 exon 20 definition and rescue ELP1 protein expression in a Familial Dysautonomia mouse model. |
| Q57803544 | Exposure to wild-type AAV drives distinct capsid immunity profiles in humans |
| Q35033532 | Extracellular metabolic energetics can promote cancer progression |
| Q38795618 | Extracellular vesicles and intercellular communication within the nervous system |
| Q57688659 | FGF21 gene therapy as treatment for obesity and insulin resistance |
| Q33782799 | Full-length dystrophin reconstitution with adeno-associated viral vectors |
| Q38772195 | Future of rAAV Gene Therapy: Platform for RNAi, Gene Editing, and Beyond |
| Q40817781 | Gene Editing for the Efficient Correction of a Recurrent COL7A1 Mutation in Recessive Dystrophic Epidermolysis Bullosa Keratinocytes. |
| Q40077177 | Gene Manipulation Strategies to Identify Molecular Regulators of Axon Regeneration in the Central Nervous System |
| Q45874307 | Gene Therapy for Hemophilia: Progress to Date. |
| Q27318007 | Gene delivery to adipose tissue using transcriptionally targeted rAAV8 vectors |
| Q38366585 | Gene therapeutic approaches to inhibit hepatitis B virus replication |
| Q90383791 | Gene therapy and genome editing for primary immunodeficiency diseases |
| Q38566097 | Gene therapy enters the pharma market: the short story of a long journey |
| Q39016569 | Gene therapy for achromatopsia. |
| Q26799437 | Gene therapy for cardiovascular disease: advances in vector development, targeting, and delivery for clinical translation |
| Q24187977 | Gene therapy for haemophilia |
| Q24202755 | Gene therapy for haemophilia |
| Q57689773 | Gene therapy for haemophilia |
| Q37903924 | Gene therapy for haemophilia: a long and winding road. |
| Q35670173 | Gene therapy for haemophilia: prospects and challenges to prevent or reverse inhibitor formation |
| Q28081911 | Gene therapy for hemophilia |
| Q42184386 | Gene therapy for hemophilia: the clot thickens. |
| Q38803022 | Gene therapy for immune tolerance induction in hemophilia with inhibitors |
| Q59340096 | Gene therapy for mucopolysaccharidoses: in vivo and ex vivo approaches |
| Q36796148 | Gene therapy for type 1 diabetes moves a step closer to reality |
| Q85807360 | Gene therapy in cystic fibrosis |
| Q38603658 | Gene therapy in monogenic congenital myopathies |
| Q38607706 | Gene therapy returns to centre stage |
| Q41820481 | Generation of Helper Plasmids Encoding Mutant Adeno-associated Virus Type 2 Capsid Proteins with Increased Resistance against Proteasomal Degradation |
| Q37043485 | Genetic Manipulation of Brown Fat Via Oral Administration of an Engineered Recombinant Adeno-associated Viral Serotype Vector |
| Q36572575 | Genetic manipulation of myoblasts and a novel primary myosatellite cell culture system: comparing and optimizing approaches |
| Q37903420 | Genetic strategies to study TDP-43 in rodents and to develop preclinical therapeutics for amyotrophic lateral sclerosis |
| Q89144123 | Genome editing by natural and engineered CRISPR-associated nucleases |
| Q38694306 | Genome editing for scalable production of alloantigen-free lentiviral vectors for in vivo gene therapy |
| Q49984648 | Genome editing technologies and their potential to treat neurologic disease |
| Q36276561 | Glycan binding avidity determines the systemic fate of adeno-associated virus type 9. |
| Q89777684 | Hemophilia gene therapy comes of age |
| Q45875635 | Hemophilia gene therapy comes of age. |
| Q42243339 | Hepatitis B virus infection enhances susceptibility toward adeno-associated viral vector transduction in vitro and in vivo. |
| Q36433979 | Hepatocyte Heparan Sulfate Is Required for Adeno-Associated Virus 2 but Dispensable for Adenovirus 5 Liver Transduction In Vivo |
| Q34629995 | High-efficiency transduction of primary human hematopoietic stem cells and erythroid lineage-restricted expression by optimized AAV6 serotype vectors in vitro and in a murine xenograft model in vivo |
| Q90196148 | Homologous Recombination-Based Genome Editing by Clade F AAVs Is Inefficient in the Absence of a Targeted DNA Break |
| Q36582838 | Host Anti-antibody Responses Following Adeno-associated Virus-mediated Delivery of Antibodies Against HIV and SIV in Rhesus Monkeys |
| Q36597262 | Human Glucocorticoid Receptor β Regulates Gluconeogenesis and Inflammation in Mouse Liver |
| Q45875226 | Human Retinal Explant Culture for Ex Vivo Validation of AAV Gene Therapy. |
| Q38183464 | Human artificial chromosome-based gene delivery vectors for biomedicine and biotechnology |
| Q35014865 | Hybrid adeno-associated viral vectors utilizing transposase-mediated somatic integration for stable transgene expression in human cells |
| Q40782473 | Hyperactive piggyBac transposons for sustained and robust liver-targeted gene therapy |
| Q36976498 | IL12-mediated liver inflammation reduces the formation of AAV transcriptionally active forms but has no effect over preexisting AAV transgene expression |
| Q90402550 | ITR-Seq, a next-generation sequencing assay, identifies genome-wide DNA editing sites in vivo following adeno-associated viral vector-mediated genome editing |
| Q37580158 | Identification of IFN-γ-producing T cells as the main mediators of the side effects associated to mouse interleukin-15 sustained exposure |
| Q40154601 | Identification of liver-specific enhancer-promoter activity in the 3' untranslated region of the wild-type AAV2 genome. |
| Q38077611 | Immune responses in liver-directed lentiviral gene therapy. |
| Q36983740 | Immune responses to AAV vectors: overcoming barriers to successful gene therapy |
| Q92093599 | Immune-orthogonal orthologues of AAV capsids and of Cas9 circumvent the immune response to the administration of gene therapy |
| Q45873745 | Immunity to CRISPR Cas9 and Cas12a therapeutics |
| Q38142098 | Immunological Monitoring to Rationally Guide AAV Gene Therapy |
| Q40928395 | Immunological ignorance allows long-term gene expression after perinatal recombinant adeno-associated virus-mediated gene transfer to murine airways |
| Q27307010 | Impact of age at administration, lysosomal storage, and transgene regulatory elements on AAV2/8-mediated rat liver transduction |
| Q40854030 | Impact of transduction towards the proliferation and migration as well as the transduction efficiency of human umbilical cord-derived late endothelial progenitor cells with nine recombinant adeno-associated virus serotypes |
| Q26800063 | Impaired vascular-mediated clearance of brain amyloid beta in Alzheimer's disease: the role, regulation and restoration of LRP1 |
| Q35451204 | Improved Immunological Tolerance Following Combination Therapy with CTLA-4/Ig and AAV-Mediated PD-L1/2 Muscle Gene Transfer. |
| Q89574758 | Improved cell-specificity of adeno-associated viral vectors for medullary thyroid carcinoma using calcitonin gene regulatory elements |
| Q40228879 | Improved gene delivery to adult mouse spinal cord through the use of engineered hybrid adeno-associated viral serotypes. |
| Q35450610 | Improving clinical efficacy of adeno associated vectors by rational capsid bioengineering. |
| Q90275153 | In Situ Modification of Tissue Stem and Progenitor Cell Genomes |
| Q39348746 | In vivo adeno-associated viral vector-mediated genetic engineering of white and brown adipose tissue in adult mice |
| Q57032336 | In vivo epigenome editing and transcriptional modulation using CRISPR technology |
| Q37045850 | In vivo gene editing in dystrophic mouse muscle and muscle stem cells |
| Q47553772 | In vivo genome editing in animals using AAV-CRISPR system: applications to translational research of human disease |
| Q34043628 | In vivo genome editing using Staphylococcus aureus Cas9 |
| Q36298651 | In vivo selection of transplanted hepatocytes by pharmacological inhibition of fumarylacetoacetate hydrolase in wild-type mice |
| Q41644107 | Incorporation of antigens into viral capsids augments immunogenicity of adeno-associated virus vector-based vaccines |
| Q27025649 | Inducing brain regeneration from within: in vivo reprogramming of endogenous somatic cells into neurons |
| Q38874623 | Induction of T-Cell Infiltration and Programmed Death Ligand 2 Expression by Adeno-Associated Virus in Rhesus Macaque Skeletal Muscle and Modulation by Prednisone |
| Q36094151 | Induction of hepatocellular carcinoma by in vivo gene targeting |
| Q55517634 | Inductively actuated micro needles for on-demand intracellular delivery. |
| Q54210526 | Influence of Pre-existing Anti-capsid Neutralizing and Binding Antibodies on AAV Vector Transduction. |
| Q37939843 | Innate Immune Responses to AAV Vectors |
| Q40875310 | Integration of AAV vectors and insertional mutagenesis |
| Q37082190 | Interception of cocaine by enzyme or antibody delivered with viral gene transfer: a novel strategy for preventing relapse in recovering drug users |
| Q45880414 | Intra-articular injection of mesenchymal stem cells expressing coagulation factor ameliorates hemophilic arthropathy in factor VIII-deficient mice |
| Q60342834 | Intracellular Delivery by Membrane Disruption: Mechanisms, Strategies, and Concepts |
| Q47549288 | Intramuscular Adeno-Associated Virus-Mediated Expression of Monoclonal Antibodies Provides 100% Protection Against Ebola Virus Infection in Mice |
| Q36114234 | Intranasal vaccination with AAV5 and 9 vectors against human papillomavirus type 16 in rhesus macaques |
| Q64244491 | Intravenous Injection of an AAV-PHP.B Vector Encoding Human Acid α-Glucosidase Rescues Both Muscle and CNS Defects in Murine Pompe Disease |
| Q51252535 | In Vitro Cellular Gene Delivery Employing a Novel Composite Material of Single-Walled Carbon Nanotubes Associated With Designed Peptides With Pegylation. |
| Q53077700 | Ionic liquids with thioether motifs as synthetic cationic lipids for gene delivery. |
| Q42203199 | Life-long correction of hyperbilirubinemia with a neonatal liver-specific AAV-mediated gene transfer in a lethal mouse model of Crigler-Najjar Syndrome |
| Q33586309 | Limitations of encapsidation of recombinant self-complementary adeno-associated viral genomes in different serotype capsids and their quantitation |
| Q35754176 | Liver production of sulfamidase reverses peripheral and ameliorates CNS pathology in mucopolysaccharidosis IIIA mice |
| Q40072904 | Local gene therapy durably restores vestibular function in a mouse model of Usher syndrome type 1G. |
| Q91636324 | Long-Term Delivery of an Anti-SIV Monoclonal Antibody With AAV |
| Q37507816 | Low-dose Gene Therapy Reduces the Frequency of Enzyme Replacement Therapy in a Mouse Model of Lysosomal Storage Disease |
| Q92796969 | Manipulation of spermatogonial stem cells in livestock species |
| Q36171849 | Mapping a neutralizing epitope onto the capsid of adeno-associated virus serotype 8. |
| Q38187651 | Mapping the AAV Capsid Host Antibody Response toward the Development of Second Generation Gene Delivery Vectors |
| Q28973610 | Massively parallel cis-regulatory analysis in the mammalian central nervous system |
| Q36939759 | MiR-298 Counteracts Mutant Androgen Receptor Toxicity in Spinal and Bulbar Muscular Atrophy |
| Q37709026 | MiR-384 inhibits human colorectal cancer metastasis by targeting KRAS and CDC42. |
| Q58768739 | MiR-384 inhibits the proliferation of colorectal cancer by targeting AKT3 |
| Q35799937 | MiRNA inhibition in tissue engineering and regenerative medicine |
| Q93055341 | MicroRNA-384 Inhibits the Progression of Papillary Thyroid Cancer by Targeting PRKACB |
| Q26747273 | MicroRNAs in glioblastoma multiforme pathogenesis and therapeutics |
| Q38012535 | Microbubble-enhanced ultrasound-mediated gene transfer--towards the development of targeted gene therapy for cancer. |
| Q44861538 | Mir-142-3p target sequences reduce transgene-directed immunogenicity following intramuscular adeno-associated virus 1 vector-mediated gene delivery |
| Q35965368 | Mitochondria-targeted antiaging gene therapy with adeno-associated viral vectors |
| Q62049234 | Modular Synthesis of Bioreducible Gene Vectors through Polyaddition of N,N′-Dimethylcystamine and Diglycidyl Ethers |
| Q41907025 | Modulation of CD8+ T cell responses to AAV vectors with IgG-derived MHC class II epitopes |
| Q39038484 | Modulation of pyridinium cationic lipid-DNA complex properties by pyridinium gemini surfactants and its impact on lipoplex transfection properties |
| Q34063834 | Molecular characterization of the newly identified human parvovirus 4 in the family Parvoviridae |
| Q37966364 | MultiBac: expanding the research toolbox for multiprotein complexes. |
| Q90704145 | Multiplexed activation of endogenous genes by CRISPRa elicits potent antitumor immunity |
| Q39352883 | Multispecies-compatible antitumor effects of a cross-species small-interfering RNA against mammalian target of rapamycin |
| Q36532822 | Muscle-directed anti-Aβ single-chain antibody delivery via AAV1 reduces cerebral Aβ load in an Alzheimer's disease mouse model |
| Q37434343 | Myotonic dystrophy mouse models: towards rational therapy development. |
| Q38690435 | Nanoscale bio-platforms for living cell interrogation: current status and future perspectives |
| Q36088172 | Near-complete elimination of mutant mtDNA by iterative or dynamic dose-controlled treatment with mtZFNs |
| Q42186779 | Neuromuscular disease. DOK7 gene therapy benefits mouse models of diseases characterized by defects in the neuromuscular junction |
| Q35117256 | Neuroprotection by rAAV-mediated gene transfer of bone morphogenic protein 7 |
| Q26801582 | New approaches to the treatment of orphan genetic disorders: Mitigating molecular pathologies using chemicals |
| Q33719424 | New directions in nicotine vaccine design and use. |
| Q38850026 | Next stop for the CRISPR revolution: RNA-guided epigenetic regulators |
| Q34668309 | Non viral vectors in gene therapy- an overview |
| Q38191018 | Non-viral gene therapy for bone tissue engineering |
| Q34429032 | Non-viral vectors for gene-based therapy |
| Q27312535 | Noninvasive and targeted gene delivery into the brain using microbubble-facilitated focused ultrasound |
| Q57172546 | Novel Approaches for the Treatment of Familial Hypercholesterolemia: Current Status and Future Challenges |
| Q39069156 | Novel anti-tumor mechanism of galanin receptor type 2 in head and neck squamous cell carcinoma cells. |
| Q46410016 | Novel approaches to render stable producer cell lines viable for the commercial manufacturing of rAAV-based gene therapy vectors |
| Q38529941 | Obstacles and future of gene therapy for hemophilia |
| Q41493938 | Off-target-free gene delivery by affinity-purified receptor-targeted viral vectors |
| Q40560600 | OneBac 2.0: Sf9 Cell Lines for Production of AAV5 Vectors with Enhanced Infectivity and Minimal Encapsidation of Foreign DNA. |
| Q37640503 | OneBac: platform for scalable and high-titer production of adeno-associated virus serotype 1-12 vectors for gene therapy |
| Q34634722 | Optimization of the capsid of recombinant adeno-associated virus 2 (AAV2) vectors: the final threshold? |
| Q40097321 | Optimized AAV rh.10 Vectors That Partially Evade Neutralizing Antibodies during Hepatic Gene Transfer |
| Q37011792 | Optimizing the transduction efficiency of capsid-modified AAV6 serotype vectors in primary human hematopoietic stem cells in vitro and in a xenograft mouse model in vivo. |
| Q36582796 | Oral-tolerization Prevents Immune Responses and Improves Transgene Persistence Following Gene Transfer Mediated by Adeno-associated Viral Vector |
| Q37691385 | Overexpression of Sarcoendoplasmic Reticulum Calcium ATPase 2a Promotes Cardiac Sympathetic Neurotransmission via Abnormal Endoplasmic Reticulum and Mitochondria Ca2+ Regulation. |
| Q30574131 | Partial correction of the CNS lysosomal storage defect in a mouse model of juvenile neuronal ceroid lipofuscinosis by neonatal CNS administration of an adeno-associated virus serotype rh.10 vector expressing the human CLN3 gene. |
| Q33649295 | Partial restoration of cardiac function with ΔPDZ nNOS in aged mdx model of Duchenne cardiomyopathy |
| Q36401085 | Patterns of scAAV vector insertion associated with oncogenic events in a mouse model for genotoxicity |
| Q38269128 | Peptide vectors for gene delivery: from single peptides to multifunctional peptide nanocarriers |
| Q38957277 | Perspective on innovative therapies for globoid cell leukodystrophy. |
| Q36085401 | Pharmacological modulation of humoral immunity in a nonhuman primate model of AAV gene transfer for hemophilia B. |
| Q40216716 | Plasmacytoid and conventional dendritic cells cooperate in crosspriming AAV capsid-specific CD8+ T cells. |
| Q37562066 | Plasmapheresis eliminates the negative impact of AAV antibodies on microdystrophin gene expression following vascular delivery. |
| Q34795284 | Portal vein delivery of viral vectors for gene therapy for hemophilia |
| Q38949089 | Pre-existing immunity to adeno-associated virus (AAV)2 limits transgene expression following intracerebral AAV2-based gene delivery in a 6-hydroxydopamine model of Parkinson's disease. |
| Q61811179 | Preclinical Development of an AAV8-hUGT1A1 Vector for the Treatment of Crigler-Najjar Syndrome |
| Q36659298 | Preclinical evaluation of an anti-HCV miRNA cluster for treatment of HCV infection |
| Q38960704 | Preferred transduction with AAV8 and AAV9 via thalamic administration in the MPS IIIB model: A comparison of four rAAV serotypes |
| Q36322970 | Prevalence and pharmacological modulation of humoral immunity to AAV vectors in gene transfer to synovial tissue |
| Q35199296 | Prevalence of anti-adeno-associated virus serotype 8 neutralizing antibodies and arylsulfatase B cross-reactive immunologic material in mucopolysaccharidosis VI patient candidates for a gene therapy trial. |
| Q38966917 | Prevalence of neutralizing antibodies against liver-tropic adeno-associated virus serotype vectors in 100 healthy Chinese and its potential relation to body constitutions |
| Q40485786 | Prevention and Reversal of Antibody Responses Against Factor IX in Gene Therapy for Hemophilia B. |
| Q39030927 | Pristimerin enhances recombinant adeno-associated virus vector-mediated transgene expression in human cell lines in vitro and murine hepatocytes in vivo |
| Q38988629 | Probing the Link among Genomic Cargo, Contact Mechanics, and Nanoindentation in Recombinant Adeno-Associated Virus 2. |
| Q50977887 | Product-Related Impurities in Clinical-Grade Recombinant AAV Vectors: Characterization and Risk Assessment. |
| Q36469478 | Proinsulin slows retinal degeneration and vision loss in the P23H rat model of retinitis pigmentosa |
| Q37621530 | Promise and problems associated with the use of recombinant AAV for the delivery of anti-HIV antibodies |
| Q41876346 | Promoterless gene targeting without nucleases rescues lethality of a Crigler-Najjar syndrome mouse model |
| Q28596492 | Proof of concept study with an HER-2 mimotope anticancer vaccine deduced from a novel AAV-mimotope library platform |
| Q39240212 | Protection against henipavirus infection by use of recombinant adeno-associated virus-vector vaccines. |
| Q61811175 | Protein-Engineered Coagulation Factors for Hemophilia Gene Therapy |
| Q35971198 | Pulmonary Targeting of Adeno-associated Viral Vectors by Next-generation Sequencing-guided Screening of Random Capsid Displayed Peptide Libraries |
| Q40596482 | RANGE: Gene Transfer of Reversibly Controlled Polycistronic Genes |
| Q37919973 | RNA-based therapeutic approaches for coagulation factor deficiencies |
| Q92244370 | RNA-guided piggyBac transposition in human cells |
| Q90109113 | RNA-guided retargeting of Sleeping Beauty transposition in human cells |
| Q38776037 | Recent Developments in Gene Therapy for Homozygous Familial Hypercholesterolemia |
| Q38907797 | Recombinant Adeno-Associated Viral Integration and Genotoxicity: Insights from Animal Models. |
| Q40084021 | Repeated AAV-mediated gene transfer by serotype switching enables long-lasting therapeutic levels of hUgt1a1 enzyme in a mouse model of Crigler-Najjar Syndrome Type I. |
| Q96292693 | Rescue of Advanced Pompe Disease in Mice with Hepatic Expression of Secretable Acid α-Glucosidase |
| Q48507292 | Rescue of GSDIII Phenotype with Gene Transfer Requires Liver- and Muscle-Targeted GDE Expression. |
| Q47301525 | Rescue of Pompe disease in mice by AAV-mediated liver delivery of secretable acid α-glucosidase. |
| Q42263324 | Rescue of amyotrophic lateral sclerosis phenotype in a mouse model by intravenous AAV9-ADAR2 delivery to motor neurons |
| Q52666370 | Retinal Cyclic Nucleotide-Gated Channels: From Pathophysiology to Therapy. |
| Q36306263 | Retinal Gene Therapy: Current Progress and Future Prospects |
| Q34044390 | Riboswitch-mediated Attenuation of Transgene Cytotoxicity Increases Adeno-associated Virus Vector Yields in HEK-293 Cells |
| Q92451499 | Ring finger protein 121 is a potent regulator of adeno-associated viral genome transcription |
| Q41923573 | Roadmap for an early gene therapy for cystic fibrosis airway disease |
| Q39428273 | Role and Therapeutic Targeting of the HGF/MET Pathway in Glioblastoma |
| Q37226788 | Role of antigen-specific regulatory CD4+CD25+ T cells in tolerance induction after neonatal IP administration of AAV-hF.IX. |
| Q21245486 | Role of the vector genome and underlying factor IX mutation in immune responses to AAV gene therapy for hemophilia B |
| Q36089404 | Safe and bodywide muscle transduction in young adult Duchenne muscular dystrophy dogs with adeno-associated virus. |
| Q91259654 | Safety and efficacy evaluations of an adeno-associated virus variant for preparing IL10-secreting human neural stem cell-based therapeutics |
| Q34487271 | Scientific overview: 2013 BBC plenary symposium on tobacco addiction |
| Q41942605 | Seeing engineered loops in a gene delivery vehicle by cryoEM. |
| Q34729878 | Selective in vivo targeting of human liver tumors by optimized AAV3 vectors in a murine xenograft model |
| Q92705326 | Senile Osteoporosis: The Involvement of Differentiation and Senescence of Bone Marrow Stromal Cells |
| Q92415298 | Sharpening the Molecular Scissors: Advances in Gene-Editing Technology |
| Q39795092 | Silencing mutant huntingtin by adeno-associated virus-mediated RNA interference ameliorates disease manifestations in the YAC128 mouse model of Huntington's disease |
| Q30583745 | Similar therapeutic efficacy between a single administration of gene therapy and multiple administrations of recombinant enzyme in a mouse model of lysosomal storage disease |
| Q41924428 | Sirtuin 6 inhibits epithelial to mesenchymal transition during idiopathic pulmonary fibrosis via inactivating TGF-β1/Smad3 signaling |
| Q36367714 | Site-Directed Mutagenesis of Surface-Exposed Lysine Residues Leads to Improved Transduction by AAV2, But Not AAV8, Vectors in Murine Hepatocytes In Vivo |
| Q92893292 | Site-Specific Glycation and Chemo-enzymatic Antibody Sortagging for the Retargeting of rAAV6 to Inflamed Endothelium |
| Q39265439 | Site-specific modification of adeno-associated viruses via a genetically engineered aldehyde tag. |
| Q38970768 | Sleeping Beauty transposition: from biology to applications. |
| Q40640594 | Smad7 gene delivery prevents muscle wasting associated with cancer cachexia in mice. |
| Q37862074 | State-of-the-art gene-based therapies: the road ahead |
| Q26825575 | State-of-the-art human gene therapy: part I. Gene delivery technologies |
| Q64993682 | Stem cells and regenerative therapies for Parkinson's disease. |
| Q33635355 | Stimuli-Responsive Mesoporous Silica NPs as Non-viral Dual siRNA/Chemotherapy Carriers for Triple Negative Breast Cancer |
| Q37056225 | Strategies for more rapid translation of cellular therapies for children: a US perspective |
| Q36398678 | Strategies to circumvent humoral immunity to adeno-associated viral vectors |
| Q27679454 | Structural Insights into Adeno-Associated Virus Serotype 5 |
| Q30152836 | Structure of neurotropic adeno-associated virus AAVrh.8. |
| Q89626645 | Structure of the AAVhu.37 capsid by cryoelectron microscopy |
| Q51739080 | Structure-activity correlation in transfection promoted by pyridinium cationic lipids. |
| Q37105696 | Studies of efficacy and liver toxicity related to adeno-associated virus-mediated RNA interference |
| Q64897549 | Supramolecular Assemblies of Peptides or Nucleopeptides for Gene Delivery. |
| Q33635361 | Sustained Inhibition of HBV Replication In Vivo after Systemic Injection of AAVs Encoding Artificial Antiviral Primary MicroRNAs |
| Q41691498 | Sustained inhibition of hepatitis B virus replication in vivo using RNAi-activating lentiviruses |
| Q40458807 | Synergistic inhibition of PARP-1 and NF-κB signaling downregulates immune response against recombinant AAV2 vectors during hepatic gene therapy. |
| Q36419827 | Synthetic CRISPR RNA-Cas9-guided genome editing in human cells |
| Q34408096 | Synthetic aptamer-polymer hybrid constructs for programmed drug delivery into specific target cells |
| Q36422224 | Synthetic riboswitches for external regulation of genes transferred by replication-deficient and oncolytic adenoviruses |
| Q28546811 | Systemic Delivery of scAAV8-Encoded MiR-29a Ameliorates Hepatic Fibrosis in Carbon Tetrachloride-Treated Mice |
| Q38644642 | Systemic gene delivery transduces the enteric nervous system of guinea pigs and cynomolgus macaques |
| Q34001776 | Systemic gene transfer reveals distinctive muscle transduction profile of tyrosine mutant AAV-1, -6, and -9 in neonatal dogs. |
| Q91645657 | TAR Cloning: Perspectives for Functional Genomics, Biomedicine, and Biotechnology |
| Q59353538 | THE JEREMIAH METZGER LECTURE NOVEL THERAPEUTIC STRATEGIES OF ALLERGIC AND IMMUNOLOGIC DISORDERS |
| Q92345066 | TPP Combined with DGUC as an Economic and Universal Process for Large-Scale Purification of AAV Vectors |
| Q42174892 | Targeted gene delivery to the enteric nervous system using AAV: a comparison across serotypes and capsid mutants. |
| Q28710390 | Targeted gene silencing to induce permanent sterility |
| Q40124877 | Targeting Visceral Fat by Intraperitoneal Delivery of Novel AAV Serotype Vector Restricting Off-Target Transduction in Liver |
| Q38110692 | Targeting long non-coding RNA to therapeutically upregulate gene expression |
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