| scholarly article | Q13442814 |
| P50 | author | Alan McClelland | Q47504204 |
| Erik D Skarsgard | Q56868047 | ||
| Bertil Glader | Q97069614 | ||
| Catherine Manno | Q43166486 | ||
| P2093 | author name string | Arthur Thompson | |
| Mark A Kay | |||
| Katherine A High | |||
| Valder R Arruda | |||
| Alan W Flake | |||
| Roland W Herzog | |||
| Margaret V Ragni | |||
| Linda B Couto | |||
| Shing Jen Tai | |||
| Ciaran Scallan | |||
| Debra G B Leonard | |||
| Margareth Ozelo | |||
| Amy J Chew | |||
| Frederick A Johnson | |||
| Peter J Larson | |||
| Sylvia Hutchison | |||
| P433 | issue | 8 | |
| P407 | language of work or name | English | Q1860 |
| P921 | main subject | hemophilia | Q134003 |
| coagulation factor IX | Q425360 | ||
| hemophilia B | Q2562598 | ||
| P1104 | number of pages | 10 | |
| P304 | page(s) | 2963-2972 | |
| P577 | publication date | 2002-12-19 | |
| P1433 | published in | Blood | Q885070 |
| P1476 | title | AAV-mediated factor IX gene transfer to skeletal muscle in patients with severe hemophilia B. | |
| P478 | volume | 101 |
| Q38937725 | 2017 Clinical trials update: Innovations in hemophilia therapy |
| Q35568505 | A 10 patient case report on the impact of plasmapheresis upon neutralizing factors against adeno-associated virus (AAV) types 1, 2, 6, and 8. |
| Q92020423 | A Molecular Revolution in the Treatment of Hemophilia |
| Q92378292 | A Practical Guide to Genome Editing Using Targeted Nuclease Technologies |
| Q34994549 | A barrel of monkeys: scAAV8 gene therapy for hemophilia in nonhuman primates. |
| Q33258821 | A look to future directions in gene therapy research for monogenic diseases |
| Q45857839 | A modified chromogenic assay for the measurement of very low levels of factor VIII activity (FVIII:C). |
| Q45879506 | A realistic chance for gene therapy in the near future |
| Q34843762 | A simple method to increase the transduction efficiency of single-stranded adeno-associated virus vectors in vitro and in vivo. |
| Q90732125 | AAV Gene Transfer with Tandem Promoter Design Prevents Anti-transgene Immunity and Provides Persistent Efficacy in Neonate Pompe Mice |
| Q92876148 | AAV Vector Immunogenicity in Humans: A Long Journey to Successful Gene Transfer |
| Q35153203 | AAV liver expression of FIX-Padua prevents and eradicates FIX inhibitor without increasing thrombogenicity in hemophilia B dogs and mice |
| Q40297313 | AAV serotype 8-mediated gene delivery of a soluble VEGF receptor to the CNS for the treatment of glioblastoma |
| Q33659389 | AAV's anatomy: roadmap for optimizing vectors for translational success |
| Q37348251 | AAV-1-mediated gene transfer to skeletal muscle in humans results in dose-dependent activation of capsid-specific T cells |
| Q37687423 | AAV-directed muscular dystrophy gene therapy |
| Q37146095 | AAV-mediated gene transfer for the treatment of hemophilia B: problems and prospects |
| Q35566831 | Adeno-associated viral vectors for retinal gene transfer. |
| Q36713767 | Adeno-associated viral vectors for the treatment of hemophilia. |
| Q40098391 | Adeno-associated virus mediated gene transfer into lung cancer cells promoting CD40 ligand-based immunotherapy |
| Q34211478 | Adeno-associated virus serotype 6 capsid tyrosine-to-phenylalanine mutations improve gene transfer to skeletal muscle |
| Q45507049 | Adeno-associated virus serotype 8 efficiently delivers genes to muscle and heart |
| Q36845715 | Adeno-associated virus vector genomes persist as episomal chromatin in primate muscle |
| Q37278165 | Adeno-associated virus vector-mediated production of hepatocyte growth factor attenuates liver fibrosis in mice |
| Q36165834 | Adeno-associated virus vectors: potential applications for cancer gene therapy |
| Q27026177 | Adeno-associated virus-mediated cancer gene therapy: current status |
| Q34317131 | Adeno-associated virus-mediated gene transfer |
| Q45457790 | Adeno-associated virus-mediated human IL-10 gene transfer suppresses the development of experimental autoimmune orchitis |
| Q34459999 | Adeno-associated virus: a key to the human genome? |
| Q27498163 | Adeno-associated virus: from defective virus to effective vector |
| Q38152792 | Adenoviruses types, cell receptors and local innate cytokines in adenovirus infection |
| Q45422040 | Administration-route and gender-independent long-term therapeutic correction of phenylketonuria (PKU) in a mouse model by recombinant adeno-associated virus 8 pseudotyped vector-mediated gene transfer |
| Q37937458 | Advancements in adeno-associated viral gene therapy approaches: exploring a new horizon |
| Q35628533 | Advances in AAV-mediated gene transfer for the treatment of inherited disorders. |
| Q34267400 | Advances in Duchenne muscular dystrophy gene therapy |
| Q35632772 | Advances in Gene Delivery Systems |
| Q36562646 | Alleviation of lung inflammatory responses by adeno-associated virus 2/9 vector carrying CC10 in OVA-sensitized mice |
| Q42215967 | Alternative strategies for gene therapy of hemophilia |
| Q37590212 | Amyotrophic lateral sclerosis (ALS): three letters that change the people's life. For ever |
| Q27012571 | Animal models of hemophilia |
| Q37091290 | Animal models of hemophilia and related bleeding disorders |
| Q36761690 | Anti-Abeta single-chain antibody delivery via adeno-associated virus for treatment of Alzheimer's disease |
| Q57022663 | Antigen-selective modulation of AAV immunogenicity with tolerogenic rapamycin nanoparticles enables successful vector re-administration |
| Q45881605 | Antigen-specific humoral tolerance or immune augmentation induced by intramuscular delivery of adeno-associated viruses encoding CTLA4-Ig-antigen fusion molecules |
| Q42425973 | Apparently nonspecific enzyme elevations after portal vein delivery of recombinant adeno-associated virus serotype 2 vector in hepatitis C virus-infected chimpanzees |
| Q33369214 | Application of a haematopoetic progenitor cell-targeted adeno-associated viral (AAV) vector established by selection of an AAV random peptide library on a leukaemia cell line |
| Q37730113 | Biodistribution of AAV8 vectors expressing human low-density lipoprotein receptor in a mouse model of homozygous familial hypercholesterolemia |
| Q38397299 | Biological therapies for inherited diseases: social and bioethical considerations. Hemophilia as an example |
| Q36437345 | Biological treatment strategies for disc degeneration: potentials and shortcomings |
| Q39122705 | Biomaterial-Guided Gene Delivery for Musculoskeletal Tissue Repair |
| Q37316887 | Birth of a new therapeutic platform: 47 years of adeno-associated virus biology from virus discovery to licensed gene therapy |
| Q35914292 | Broadly Neutralizing Human Immunodeficiency Virus Type 1 Antibody Gene Transfer Protects Nonhuman Primates from Mucosal Simian-Human Immunodeficiency Virus Infection |
| Q38784208 | CRISPR/Cas9-mediated somatic and germline gene correction to restore hemostasis in hemophilia B mice. |
| Q37095348 | Cardiovascular toxicity and titin cross-reactivity of affinity-enhanced T cells in myeloma and melanoma. |
| Q33938435 | Cell-Mediated Immunity to AAV Vectors, Evolving Concepts and Potential Solutions |
| Q38787435 | Characterization of the Adeno-Associated Virus 1 and 6 Sialic Acid Binding Site |
| Q91963191 | Chimeric Capsid Proteins Impact Transduction Efficiency of Haploid Adeno-Associated Virus Vectors |
| Q26743393 | Clinical development of gene therapy: results and lessons from recent successes |
| Q37140649 | Clinical gene therapy using recombinant adeno-associated virus vectors |
| Q43978844 | Clinical trials and SCID row: the ethics of phase 1 trials in the developing world |
| Q42256024 | Coagulation Factor IX for Hemophilia B Therapy |
| Q45864237 | Coaxing coagulation with RNA and cell fragments |
| Q33797482 | Comparative analysis of virus-host interactions caused by a virulent and an attenuated duck hepatitis A virus genotype 1 |
| Q33832790 | Comparison of adeno-associated virus pseudotype 1, 2, and 8 vectors administered by intramuscular injection in the treatment of murine phenylketonuria |
| Q87942816 | Complete correction of hemophilia B phenotype by FIX-Padua skeletal muscle gene therapy in an inhibitor-prone dog model |
| Q39394803 | Complexity of immune responses to AAV transgene products - Example of factor IX. |
| Q40150114 | Construction of diverse adeno-associated viral libraries for directed evolution of enhanced gene delivery vehicles |
| Q28072423 | Current animal models of hemophilia: the state of the art |
| Q45856057 | Current issues facing coagulationists--meeting report |
| Q35881934 | Current status and prospects for gene therapy |
| Q47626470 | DNA and low titer, helper-free, recombinant AAV prime-boost vaccination for cytomegalovirus induces an immune response to CMV-pp65 and CMV-IE1 in transgenic HLA A*0201 mice |
| Q43055175 | Death switch for gene therapy: application to erythropoietin transgene expression |
| Q37299072 | Designing heart performance by gene transfer. |
| Q28730144 | Development and utility of an internal threshold control (ITC) real-time PCR assay for exogenous DNA detection |
| Q38069585 | Development of Novel Recombinant AAV Vectors and Strategies for the Potential Gene Therapy of Hemophilia |
| Q30152887 | Development of Patient-specific AAV Vectors After Neutralizing Antibody Selection for Enhanced Muscle Gene Transfer |
| Q35184217 | Developmental stage determines efficiency of gene transfer to muscle satellite cells by in utero delivery of adeno-associated virus vector serotype 2/9 |
| Q26823376 | Developments in the treatment of hemophilia B: focus on emerging gene therapy |
| Q33521174 | Directed evolution of a novel adeno-associated virus (AAV) vector that crosses the seizure-compromised blood-brain barrier (BBB). |
| Q33232270 | Directed evolution of adeno-associated virus yields enhanced gene delivery vectors |
| Q37385999 | Distinct immune responses to transgene products from rAAV1 and rAAV8 vectors |
| Q42667110 | Diverse IgG subclass responses to adeno-associated virus infection and vector administration |
| Q64046774 | Drug discovery and development for rare genetic disorders |
| Q34183415 | Duration of expression and activity of Sleeping Beauty transposase in mouse liver following hydrodynamic DNA delivery |
| Q39031877 | Effect and mechanism of Mitomycin C combined with recombinant adeno-associated virus type II against glioma |
| Q37226783 | Effect of vascular endothelial growth factor gene therapy on post-traumatic peripheral nerve regeneration and denervation-related muscle atrophy |
| Q45872187 | Effect of viral dose on neutralizing antibody response and transgene expression after AAV1 vector re-administration in mice |
| Q41999615 | Effects of transient immunosuppression on adenoassociated, virus-mediated, liver-directed gene transfer in rhesus macaques and implications for human gene therapy |
| Q37925857 | Efficacy and safety of Sleeping Beauty transposon-mediated gene transfer in preclinical animal studies |
| Q42252699 | Efficient AAV1-AAV2 hybrid vector for gene therapy of hemophilia |
| Q45428790 | Efficient hepatic delivery and expression from a recombinant adeno-associated virus 8 pseudotyped alpha1-antitrypsin vector |
| Q36960845 | Emerging strategies for cell and gene therapy of the muscular dystrophies |
| Q42245041 | Encapsulation of factor IX-engineered mesenchymal stem cells in fibrinogen-alginate microcapsules enhances their viability and transgene secretion |
| Q37982239 | Endothelial progenitor cell-based therapy for hemophilia A. |
| Q40532563 | Engagement of the B-cell antigen receptor (BCR) allows efficient transduction of ZAP-70-positive primary B-CLL cells by recombinant adeno-associated virus (rAAV) vectors |
| Q36737263 | Engineering Factor Viii for Hemophilia Gene Therapy |
| Q37466675 | Enhanced factor VIII heavy chain for gene therapy of hemophilia A |
| Q41881145 | Enhanced long-term transduction and multilineage engraftment of human hematopoietic stem cells transduced with tyrosine-modified recombinant adeno-associated virus serotype 2. |
| Q35635046 | Enhanced selective gene delivery to neural stem cells in vivo by an adeno-associated viral variant |
| Q39595690 | Enhancers of adeno-associated virus AAV2 transduction via high throughput siRNA screening |
| Q35194907 | Enhancing gene delivery of adeno-associated viruses by cell-permeable peptides |
| Q37042394 | Enhancing transduction of the liver by adeno-associated viral vectors |
| Q34541550 | Eradication of neutralizing antibodies to factor VIII in canine hemophilia A after liver gene therapy |
| Q40986812 | Establishment of two quantitative nested qPCR assays targeting the human EPO transgene. |
| Q45859498 | Evidence of multiyear factor IX expression by AAV-mediated gene transfer to skeletal muscle in an individual with severe hemophilia B. |
| Q36715322 | Extracorporeal delivery of rAAV with metabolic exchange and oxygenation |
| Q34122653 | Factor IX ectopically expressed in platelets can be stored in alpha-granules and corrects the phenotype of hemophilia B mice |
| Q35876970 | Factor IX expression in skeletal muscle of a severe hemophilia B patient 10 years after AAV-mediated gene transfer |
| Q37387091 | Factor VIII inhibitors: risk factors and methods for prevention and immune modulation |
| Q51057724 | Gene Therapy for Hemophilia. |
| Q99579530 | Gene Therapy for Hemophilia: Facts and Quandaries in the 21st Century |
| Q45874307 | Gene Therapy for Hemophilia: Progress to Date. |
| Q47225946 | Gene Therapy with BMN 270 Results in Therapeutic Levels of FVIII in Mice and Primates and Normalization of Bleeding in Hemophilic Mice |
| Q45859041 | Gene doping detection: evaluation of approach for direct detection of gene transfer using erythropoietin as a model system |
| Q33691825 | Gene expression in lung and liver after intravenous infusion of polyethylenimine complexes of Sleeping Beauty transposons |
| Q34566454 | Gene replacement therapies for duchenne muscular dystrophy using adeno-associated viral vectors |
| Q38260248 | Gene replacement therapy for genetic hepatocellular jaundice |
| Q57677438 | Gene therapy for Rett syndrome: prospects and challenges |
| Q36800517 | Gene therapy for cardiac arrhythmias: a dream soon to come true? |
| Q36816069 | Gene therapy for disorders affecting children, progress and potential |
| Q24187977 | Gene therapy for haemophilia |
| Q24202755 | Gene therapy for haemophilia |
| Q57689773 | Gene therapy for haemophilia |
| Q80702723 | Gene therapy for haemophilia |
| Q45879900 | Gene therapy for haemophilia B. |
| Q37903924 | Gene therapy for haemophilia: a long and winding road. |
| Q28081911 | Gene therapy for hemophilia |
| Q90204599 | Gene therapy for hemophilia: Progress to date and challenges moving forward |
| Q42184386 | Gene therapy for hemophilia: the clot thickens. |
| Q57167448 | Gene therapy for hemophilia: what does the future hold? |
| Q33881920 | Gene therapy for monogenic liver diseases: clinical successes, current challenges and future prospects. |
| Q24680426 | Gene therapy for severe combined immunodeficiency: are we there yet? |
| Q36446948 | Gene therapy for treatment of inherited haematological disorders |
| Q28087557 | Gene therapy in an era of emerging treatment options for hemophilia B |
| Q35630146 | Gene therapy in orthopaedic surgery: the current status |
| Q40565579 | Gene therapy platform for bone regeneration using an exogenously regulated, AAV-2-based gene expression system. |
| Q35712206 | Gene therapy progress and prospects: recombinant adeno-associated virus (rAAV) vectors |
| Q36345325 | Gene therapy strategies for Duchenne muscular dystrophy utilizing recombinant adeno-associated virus vectors. |
| Q37789715 | Gene therapy strategies for hemophilia: benefits versus risks |
| Q34852719 | Gene therapy using adeno-associated virus vectors |
| Q40258000 | Gene transfer as a strategy to achieve permanent cardioprotection I: rAAV-mediated gene therapy with inducible nitric oxide synthase limits infarct size 1 year later without adverse functional consequences |
| Q40257549 | Gene transfer as a strategy to achieve permanent cardioprotection II: rAAV-mediated gene therapy with heme oxygenase-1 limits infarct size 1 year later without adverse functional consequences |
| Q36230845 | Gene transfer for hemophilia: can therapeutic efficacy in large animals be safely translated to patients? |
| Q37684597 | Gene transfer of arginine kinase to skeletal muscle using adeno-associated virus |
| Q35571114 | Generation and in vivo evaluation of IL10-treated dendritic cells in a nonhuman primate model of AAV-based gene transfer |
| Q43056999 | Generation of a novel factor IX with augmented clotting activities in vitro and in vivo. |
| Q34843808 | Good manufacturing practice production of self-complementary serotype 8 adeno-associated viral vector for a hemophilia B clinical trial |
| Q35143113 | Haemophilias A and B. |
| Q45869156 | Has gene therapy for ALS arrived? |
| Q36004744 | Heat-shock treatment-mediated increase in transduction by recombinant adeno-associated virus 2 vectors is independent of the cellular heat-shock protein 90 |
| Q89777684 | Hemophilia gene therapy comes of age |
| Q45875635 | Hemophilia gene therapy comes of age. |
| Q40726384 | Hepatocyte growth factor receptor is a coreceptor for adeno-associated virus type 2 infection. |
| Q34382965 | High-efficiency transduction and correction of murine hemophilia B using AAV2 vectors devoid of multiple surface-exposed tyrosines. |
| Q34329364 | High-efficiency transduction of fibroblasts and mesenchymal stem cells by tyrosine-mutant AAV2 vectors for their potential use in cellular therapy |
| Q33863855 | Hoechst increases adeno-associated virus-mediated transgene expression in airway epithelia by inducing the cytomegalovirus promoter |
| Q33713716 | Host and vector-dependent effects on the risk of germline transmission of AAV vectors |
| Q37406656 | Human studies of angiogenic gene therapy |
| Q37236099 | Humoral Immune Response to AAV. |
| Q45446011 | IFN-alpha gene therapy for woodchuck hepatitis with adeno-associated virus: differences in duration of gene expression and antiviral activity using intraportal or intramuscular routes |
| Q42183295 | Immune deviation by mucosal antigen administration suppresses gene-transfer-induced inhibitor formation to factor IX. |
| Q45866729 | Immune responses to AAV capsid: are mice not humans after all? |
| Q36983740 | Immune responses to AAV vectors: overcoming barriers to successful gene therapy |
| Q38287263 | Immune responses to intramuscular administration of alipogene tiparvovec (AAV1-LPL(S447X)) in a phase II clinical trial of lipoprotein lipase deficiency gene therapy |
| Q37943306 | Immunity and AAV-Mediated Gene Therapy for Muscular Dystrophies in Large Animal Models and Human Trials |
| Q35575706 | Impact of pre-existing immunity on gene transfer to nonhuman primate liver with adeno-associated virus 8 vectors |
| Q39501844 | Impact of the underlying mutation and the route of vector administration on immune responses to factor IX in gene therapy for hemophilia B. |
| Q34741419 | In utero therapy for congenital disorders using amniotic fluid stem cells |
| Q38824171 | In vivo tissue-tropism of adeno-associated viral vectors |
| Q44328165 | Inadvertent germline transmission of AAV2 vector: findings in a rabbit model correlate with those in a human clinical trial |
| Q39506153 | Induction of immune tolerance to FIX following muscular AAV gene transfer is AAV-dose/FIX-level dependent |
| Q35186003 | Induction of robust immune responses against human immunodeficiency virus is supported by the inherent tropism of adeno-associated virus type 5 for dendritic cells |
| Q54210526 | Influence of Pre-existing Anti-capsid Neutralizing and Binding Antibodies on AAV Vector Transduction. |
| Q35734826 | Intracellular transport of recombinant adeno-associated virus vectors |
| Q40487347 | Intramuscular administration of AAV overcomes pre-existing neutralizing antibodies in rhesus macaques. |
| Q35418068 | Intramuscular injection of AAV8 in mice and macaques is associated with substantial hepatic targeting and transgene expression |
| Q40488137 | Intrapleural administration of a serotype 5 adeno-associated virus coding for alpha1-antitrypsin mediates persistent, high lung and serum levels of alpha1-antitrypsin |
| Q37524906 | K137R mutation on adeno-associated viral capsids had minimal effect on enhancing gene delivery in vivo |
| Q34467839 | LDLR-Gene therapy for familial hypercholesterolaemia: problems, progress, and perspectives |
| Q47311266 | LOW HOX gene expression in PML-RARα-POSITIVE leukemia results from suppressed histone demethylation. |
| Q39639059 | Lack of immunotoxicity after regional intravenous (RI) delivery of rAAV to nonhuman primate skeletal muscle |
| Q30409863 | Lessons Learned from Animal Models of Inherited Bleeding Disorders |
| Q43268180 | Limb-girdle muscular dystrophy type 2D gene therapy restores alpha-sarcoglycan and associated proteins |
| Q39553052 | Liver-Directed Adeno-Associated Viral Gene Therapy for Hemophilia |
| Q36940126 | Liver-specific microRNA-122 target sequences incorporated in AAV vectors efficiently inhibits transgene expression in the liver |
| Q44666833 | Local gene knockdown in the brain using viral-mediated RNA interference. |
| Q96591115 | Long-Term Follow-Up of the First in Human Intravascular Delivery of AAV for Gene Transfer: AAV2-hFIX16 for Severe Hemophilia B |
| Q37071092 | Long-term correction of inhibitor-prone hemophilia B dogs treated with liver-directed AAV2-mediated factor IX gene therapy |
| Q45858191 | Long-term expression and repeated administration of AAV type 1, 2 and 5 vectors in skeletal muscle of immunocompetent adult mice |
| Q28589503 | Long-term rescue of a lethal inherited disease by adeno-associated virus-mediated gene transfer in a mouse model of molybdenum-cofactor deficiency |
| Q34994506 | Long-term safety and efficacy following systemic administration of a self-complementary AAV vector encoding human FIX pseudotyped with serotype 5 and 8 capsid proteins. |
| Q34783922 | Long-term safety and efficacy of factor IX gene therapy in hemophilia B. |
| Q45868167 | Longevity of rAAV vector and plasmid DNA in blood after intramuscular injection in nonhuman primates: implications for gene doping |
| Q45883516 | Major role of local immune responses in antibody formation to factor IX in AAV gene transfer |
| Q26750799 | Manufacturing of recombinant adeno-associated viral vectors for clinical trials |
| Q37932009 | MeCP2 and Rett syndrome: reversibility and potential avenues for therapy. |
| Q38081027 | Mechanistic, technical, and clinical perspectives in therapeutic stimulation of coronary collateral development by angiogenic growth factors |
| Q38844559 | Mesothelin-specific cell-based vaccine generates antigen-specific immunity and potent antitumor effects by combining with IL-12 immunomodulator. |
| Q41318528 | Minimizing the inhibitory effect of neutralizing antibody for efficient gene expression in the liver with adeno-associated virus 8 vectors. |
| Q44861538 | Mir-142-3p target sequences reduce transgene-directed immunogenicity following intramuscular adeno-associated virus 1 vector-mediated gene delivery |
| Q40055959 | Muscle Gene Therapy for Hemophilia. |
| Q41136552 | Muscle function recovery in golden retriever muscular dystrophy after AAV1-U7 exon skipping. |
| Q36085709 | Muscle-directed gene therapy for hemophilia B with more efficient and less immunogenic AAV vectors. |
| Q39519818 | Neutralizing antibodies against adeno-associated virus examined prospectively in pediatric patients with hemophilia |
| Q40354609 | Neutralizing antibodies against adeno-associated viruses in Sjögren's patients: implications for gene therapy |
| Q38622440 | New and improved AAVenues: current status of hemophilia B gene therapy |
| Q44815512 | Non-invasive viral gene transfer of factor IX to colonic epithelial cells in hemophilia B mice |
| Q42627671 | Noninvasive repetitive imaging of somatostatin receptor 2 gene transfer with positron emission tomography. |
| Q37743813 | Novel adeno-associated virus vector vaccine restricts replication of simian immunodeficiency virus in macaques |
| Q46410016 | Novel approaches to render stable producer cell lines viable for the commercial manufacturing of rAAV-based gene therapy vectors |
| Q34228552 | Novel caprine adeno-associated virus (AAV) capsid (AAV-Go.1) is closely related to the primate AAV-5 and has unique tropism and neutralization properties |
| Q38529941 | Obstacles and future of gene therapy for hemophilia |
| Q50333514 | Oligonucleotide conjugated multi-functional adeno-associated viruses. |
| Q42092752 | Optimal Immunofluorescent Staining for Human Factor IX and Infiltrating T Cells following Gene Therapy for Hemophilia B. |
| Q33832799 | Optimized adeno-associated virus (AAV)-protein phosphatase-5 helper viruses for efficient liver transduction by single-stranded AAV vectors: therapeutic expression of factor IX at reduced vector doses |
| Q64092258 | Oracle or false prophet? Can we predict AAV efficacy based on preexisting antibody titers? |
| Q42228305 | Oral gene therapy for hemophilia B using chitosan-formulated FIX mutants |
| Q36582796 | Oral-tolerization Prevents Immune Responses and Improves Transgene Persistence Following Gene Transfer Mediated by Adeno-associated Viral Vector |
| Q38562869 | Our journey to successful gene therapy for hemophilia B. |
| Q42056009 | Overcoming preexisting humoral immunity to AAV using capsid decoys |
| Q34361637 | PCR-based detection of gene transfer vectors: application to gene doping surveillance |
| Q26851325 | Parkinson's disease gene therapy: success by design meets failure by efficacy |
| Q52100726 | Peers rally in support of accused scientist. |
| Q45855280 | Perioperative haemostatic management of haemophilic mice using normal mouse plasma |
| Q33931862 | Peripheral transvenular delivery of adeno-associated viral vectors to skeletal muscle as a novel therapy for hemophilia B. |
| Q45868785 | Persistent expression of hF.IX After tolerance induction by in utero or neonatal administration of AAV-1-F.IX in hemophilia B mice |
| Q36293299 | Pharmacokinetics of plasmid DNA-based non-viral gene medicine |
| Q36085401 | Pharmacological modulation of humoral immunity in a nonhuman primate model of AAV gene transfer for hemophilia B. |
| Q35496285 | Phase 2 clinical trial of a recombinant adeno-associated viral vector expressing α1-antitrypsin: interim results |
| Q51143641 | Phenotypic Correction of α-Sarcoglycan Deficiency by Intra-arterial Injection of a Muscle-specific Serotype 1 rAAV Vector. |
| Q34994501 | Phenotypic correction of a mouse model for primary hyperoxaluria with adeno-associated virus gene transfer |
| Q40385653 | Phenotypic improvement of dystrophic muscles by rAAV/microdystrophin vectors is augmented by Igf1 codelivery. |
| Q38012709 | Phoenix rising: gene therapy makes a comeback |
| Q37801839 | Physiological and tissue-specific vectors for treatment of inherited diseases |
| Q37562066 | Plasmapheresis eliminates the negative impact of AAV antibodies on microdystrophin gene expression following vascular delivery. |
| Q37993976 | Platelet and endothelial expression of clotting factors for the treatment of hemophilia |
| Q34348837 | Platelets as delivery systems for disease treatments |
| Q36902844 | Potential use of gene transfer in athletic performance enhancement. |
| Q36302317 | Precise hit: adeno-associated virus in gene targeting |
| Q34589262 | Preclinical Evaluation of a Recombinant Adeno-Associated Virus Vector Expressing Human Alpha-1 Antitrypsin Made Using a Recombinant Herpes Simplex Virus Production Method |
| Q35086714 | Preclinical differences of intravascular AAV9 delivery to neurons and glia: a comparative study of adult mice and nonhuman primates |
| Q45874908 | Preclinical safety evaluation of subretinal AAV2.sFlt-1 in non-human primates. |
| Q37760380 | Preclinical studies for gene therapy of Duchenne muscular dystrophy. |
| Q36322970 | Prevalence and pharmacological modulation of humoral immunity to AAV vectors in gene transfer to synovial tissue |
| Q40485786 | Prevention and Reversal of Antibody Responses Against Factor IX in Gene Therapy for Hemophilia B. |
| Q34574858 | Prevention of cytotoxic T lymphocyte responses to factor IX-expressing hepatocytes by gene transfer-induced regulatory T cells |
| Q39062624 | Production of adeno-associated virus (AAV) serotypes by transient transfection of HEK293 cell suspension cultures for gene delivery |
| Q36713761 | Progress and challenges in viral vector manufacturing |
| Q33844184 | Progress towards gene therapy for haemophilia B. |
| Q38981274 | Prolonged expression of an anti-HIV-1 gp120 minibody to the female rhesus macaque lower genital tract by AAV gene transfer |
| Q37621530 | Promise and problems associated with the use of recombinant AAV for the delivery of anti-HIV antibodies |
| Q35824841 | Prospects for gene therapy of haemophilia |
| Q28730577 | Proteasome inhibition is partially effective in attenuating pre-existing immunity against recombinant adeno-associated viral vectors |
| Q35006603 | Protein replacement therapy and gene transfer in canine models of hemophilia A, hemophilia B, von willebrand disease, and factor VII deficiency |
| Q40179808 | Rapamycin-regulated control of antiangiogenic tumor therapy following rAAV-mediated gene transfer |
| Q38843434 | Recent tissue engineering-based advances for effective rAAV-mediated gene transfer in the musculoskeletal system |
| Q43502862 | Recombinant AAV2-mediated β-globin expression in human fetal hematopoietic cells from the aborted fetuses with β-thalassemia major |
| Q91707074 | Recombinant Adeno-Associated Virus Gene Therapy in Light of Luxturna (and Zolgensma and Glybera): Where Are We, and How Did We Get Here? |
| Q37731874 | Recombinant Adeno-Associated Virus Vector Genomes Take the Form of Long-Lived, Transcriptionally Competent Episomes in Human Muscle |
| Q35904461 | Recombinant adeno-associated viral (rAAV) vectors as therapeutic tools for Duchenne muscular dystrophy (DMD). |
| Q34706818 | Recombinant adeno-associated virus serotype 6 efficiently transduces primary human melanocytes |
| Q36144935 | Recombinant adeno-associated virus vectors induce functionally impaired transgene product-specific CD8+ T cells in mice |
| Q45370683 | Recombinant adeno-associated virus-mediated gene transfer for the potential therapy of adenosine deaminase-deficient severe combined immune deficiency. |
| Q35847459 | Regional intravascular delivery of AAV-2-F.IX to skeletal muscle achieves long-term correction of hemophilia B in a large animal model |
| Q40282843 | Regulatory and Exhausted T Cell Responses to AAV Capsid. |
| Q47301525 | Rescue of Pompe disease in mice by AAV-mediated liver delivery of secretable acid α-glucosidase. |
| Q40344567 | Restoration of fatty aldehyde dehydrogenase deficiency in Sjögren-Larsson syndrome |
| Q21245486 | Role of the vector genome and underlying factor IX mutation in immune responses to AAV gene therapy for hemophilia B |
| Q54457954 | Safety and Efficacy of Regional Intravenous (RI) Versus Intramuscular (IM) Delivery of rAAV1 and rAAV8 to Nonhuman Primate Skeletal Muscle. |
| Q34700126 | Safety and effects of the vector for the Leber hereditary optic neuropathy gene therapy clinical trial. |
| Q36438711 | 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. |
| Q40444722 | Safety characterization of HeLa-based cell substrates used in the manufacture of a recombinant adeno-associated virus-HIV vaccine |
| Q34025098 | Safety of AAV factor IX peripheral transvenular gene delivery to muscle in hemophilia B dogs. |
| Q35101904 | Safety of liver gene transfer following peripheral intravascular delivery of adeno-associated virus (AAV)-5 and AAV-6 in a large animal model |
| Q38168940 | Safety profile of recombinant adeno-associated viral vectors: focus on alipogene tiparvovec (Glybera®). |
| Q35431994 | Self-complementary adeno-associated viral vectors for gene therapy of hemophilia B: progress and challenges |
| Q40436594 | Serotype-specific replicating AAV helper constructs increase recombinant AAV type 2 vector production |
| Q36171820 | Single amino acid modification of adeno-associated virus capsid changes transduction and humoral immune profiles |
| Q40619677 | Single tyrosine mutation in AAV8 and AAV9 capsids is insufficient to enhance gene delivery to skeletal muscle and heart |
| Q39265439 | Site-specific modification of adeno-associated viruses via a genetically engineered aldehyde tag. |
| Q51759036 | Small Molecule Catalysts with Therapeutic Potential. |
| Q45873385 | Specific detection of human coagulation factor IX in cynomolgus macaques |
| Q40434248 | Stable antibody expression at therapeutic levels using the 2A peptide |
| Q37862074 | State-of-the-art gene-based therapies: the road ahead |
| Q37001594 | Staunch protections: the ethics of haemophilia gene transfer research |
| Q39979752 | Strategies for improving the transduction efficiency of single-stranded adeno-associated virus vectors in vitro and in vivo |
| Q36398678 | Strategies to circumvent humoral immunity to adeno-associated viral vectors |
| Q33713999 | Strategies to modulate immune responses: a new frontier for gene therapy |
| Q40581112 | Striatal delivery of rAAV-hAADC to rats with preexisting immunity to AAV. |
| Q29619095 | Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response |
| Q36341646 | Super-resolution imaging of nuclear import of adeno-associated virus in live cells |
| Q45457856 | Sustained correction of glycogen storage disease type II using adeno-associated virus serotype 1 vectors |
| Q37695999 | Systemic delivery of factor IX messenger RNA for protein replacement therapy |
| Q34347199 | Systemic delivery of genes to striated muscles using adeno-associated viral vectors. |
| Q35847626 | Systemic protein delivery by muscle-gene transfer is limited by a local immune response |
| Q42400718 | TLR3 signaling does not affect organ-specific immune responses to factor IX in AAV gene therapy |
| Q45876374 | The Genetics of bleeding disorders: a report on the UK Haemophilia Centre Doctors' Organisation annual scientific symposium, 10th October 2003. |
| Q94464382 | The Immune Response to the fVIII Gene Therapy in Preclinical Models |
| Q37348229 | The Jeremiah Metzger Lecture: gene therapy for inherited disorders: from Christmas disease to Leber's amaurosis |
| Q38539938 | The Skeletal Muscle Environment and Its Role in Immunity and Tolerance to AAV Vector-Mediated Gene Transfer |
| Q36440058 | The efficacy and the risk of immunogenicity of FIX Padua (R338L) in hemophilia B dogs treated by AAV muscle gene therapy. |
| Q26823818 | The gene therapy journey for hemophilia: are we there yet? |
| Q35559616 | The hopes and fears of in utero gene therapy for genetic disease--a review. |
| Q40361106 | The inhibitory effects of anticoagulation on in vivo gene transfer by adeno-associated viral or adenoviral vectors |
| Q26751281 | The interplay of post-translational modification and gene therapy |
| Q33902499 | The potential of adeno-associated viral vectors for gene delivery to muscle tissue |
| Q42261520 | The prevalence of neutralizing antibodies against adeno-associated virus capsids is reduced in young Japanese individuals |
| Q35613460 | The promise of third-generation recombinant therapy and gene therapy |
| Q45166575 | The role of immunosuppression in gene- and cell-based treatments for duchenne muscular dystrophy |
| Q37120438 | The role of the adeno-associated virus capsid in gene transfer |
| Q34982869 | Therapeutic approaches to muscular dystrophy. |
| Q37866086 | Therapeutic in vivo gene transfer for genetic disease using AAV: progress and challenges |
| Q36066048 | Therapy for haemophilia: recent advances and goals for the future |
| Q96109656 | Timing of Intensive Immunosuppression Impacts Risk of Transgene Antibodies after AAV Gene Therapy in Nonhuman Primates |
| Q36320026 | Tolerance induction by viral in vivo gene transfer |
| Q36068277 | Towards safe, non-viral therapeutic gene expression in humans. |
| Q30882233 | Translational Data from AAV-Mediated Gene Therapy of Hemophilia B in Dogs |
| Q30891500 | Translational data from adeno-associated virus-mediated gene therapy of hemophilia B in dogs |
| Q30539199 | Treatment of diabetes and long-term survival after insulin and glucokinase gene therapy. |
| Q36448393 | Treatment of human disease by adeno-associated viral gene transfer |
| Q37181630 | Ultrasound‑targeted microbubble destruction enhances gene transduction of adeno-associated virus in a less-permissive cell type, NIH/3T3. |
| Q37768868 | Unique strategies for therapeutic gene transfer in haemophilia A and haemophilia BWFH State-of-the-Art Session on Therapeutic Gene Transfer Buenos Aires, Argentina |
| Q44804864 | Update on gene therapy for hereditary hematological disorders |
| Q37024699 | Using homologous recombination to manipulate the genome of human somatic cells |
| Q60307880 | Vectored delivery of anti-SIV envelope targeting mAb via AAV8 protects rhesus macaques from repeated limiting dose intrarectal swarm SIVsmE660 challenge |
| Q34471754 | Viral-based modelling and correction of neurodegenerative diseases by RNA interference. |
| Q35847178 | Viral-mediated gene therapy for the muscular dystrophies: successes, limitations and recent advances |
| Q34313224 | Virus-delivered small RNA silencing sustains strength in amyotrophic lateral sclerosis |
| Q35915700 | What is a cure and how do we get there? |
Search more.