| scholarly article | Q13442814 |
| P50 | author | Simon N. Waddington | Q37835224 |
| Katherine A. High | Q41502889 | ||
| Federico Mingozzi | Q89436980 | ||
| Andrew M Davidoff | Q114449144 | ||
| P2093 | author name string | Cecilia Rosales | |
| Roderick Bronson | |||
| Arthur W Nienhuis | |||
| M Beth McCarville | |||
| Robert E Donahue | |||
| Amit C Nathwani | |||
| Scott Jackson | |||
| John T Gray | |||
| Catherine Y C Ng | |||
| Junfang Zhou | |||
| Yunyu Spence | |||
| Jenny McIntosh | |||
| Marc Valentine | |||
| Deepak Raj | |||
| John Coleman | |||
| Susan Sleep | |||
| Devhrut Nathwani | |||
| Ghasem Rastegarlari | |||
| James Allay | |||
| Sushmita Nawathe | |||
| P2860 | cites work | Latent adeno-associated virus infection elicits humoral but not cell-mediated immune responses in a nonhuman primate model | Q24527302 |
| Novel adeno-associated viruses from rhesus monkeys as vectors for human gene therapy | Q24535690 | ||
| Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response | Q29619095 | ||
| The pleiotropic effects of natural AAV infections on liver-directed gene transfer in macaques | Q33730459 | ||
| Large-scale molecular characterization of adeno-associated virus vector integration in mouse liver | Q33737640 | ||
| Extrachromosomal recombinant adeno-associated virus vector genomes are primarily responsible for stable liver transduction in vivo | Q33844362 | ||
| Chromatin silencing and the maintenance of a functional germline in Caenorhabditis elegans | Q33855919 | ||
| Diversity of homology-dependent gene silencing strategies in fungi | Q33879824 | ||
| Peripheral transvenular delivery of adeno-associated viral vectors to skeletal muscle as a novel therapy for hemophilia B. | Q33931862 | ||
| Neonatal intravenous injection of a gammaretroviral vector has a low incidence of tumor induction in mice | Q33936562 | ||
| Characterization of adeno-associated virus genomes isolated from human tissues | Q34142848 | ||
| AAV-mediated factor IX gene transfer to skeletal muscle in patients with severe hemophilia B. | Q34168942 | ||
| Canine hemophilia B resulting from a point mutation with unusual consequences | Q34326780 | ||
| Inactivation of gene expression in plants as a consequence of specific sequence duplication | Q35179456 | ||
| 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 | ||
| Prospects for gene therapy of haemophilia | Q35824841 | ||
| 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 | ||
| Current status and prospects for gene therapy | Q35881934 | ||
| Modulation of tolerance to the transgene product in a nonhuman primate model of AAV-mediated gene transfer to liver. | Q36007714 | ||
| 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 | ||
| Inhibitor development in haemophilia B: an orphan disease in need of attention | Q36872038 | ||
| Enhancing transduction of the liver by adeno-associated viral vectors | Q37042394 | ||
| Manufacturing and characterizing AAV-based vectors for use in clinical studies | Q37140657 | ||
| Unrestricted hepatocyte transduction with adeno-associated virus serotype 8 vectors in mice | Q37747148 | ||
| Comparison of the ability of adeno-associated viral vectors pseudotyped with serotype 2, 5, and 8 capsid proteins to mediate efficient transduction of the liver in murine and nonhuman primate models | Q40418515 | ||
| Infectious titer assay for adeno-associated virus vectors with sensitivity sufficient to detect single infectious events | Q40527772 | ||
| The morphology, immunohistochemistry, and incidence of hematopoietic neoplasms in mice and rats | Q40766801 | ||
| Targeted inactivation of the coagulation factor IX gene causes hemophilia B in mice. | Q43169559 | ||
| Repeat-induced gene silencing in mammals. | Q44031048 | ||
| Lifespan, age changes and tumour incidence in an ageing C57BL mouse colony | Q44038322 | ||
| Permanent phenotypic correction of hemophilia B in immunocompetent mice by prenatal gene therapy. | Q44220504 | ||
| Efficient gene transfer into human cord blood CD34+ cells and the CD34+CD38- subset using highly purified recombinant adeno-associated viral vector preparations that are free of helper virus and wild-type AAV. | Q45151455 | ||
| Analysis of tumors arising in male B6C3F1 mice with and without AAV vector delivery to liver. | Q45225518 | ||
| Heparin binding directs activation of T cells against adeno-associated virus serotype 2 capsid | Q45414481 | ||
| Evidence for repeat-induced gene silencing in cultured Mammalian cells: inactivation of tandem repeats of transfected genes | Q45713496 | ||
| Sustained high-level expression of human factor IX (hFIX) after liver-targeted delivery of recombinant adeno-associated virus encoding the hFIX gene in rhesus macaques | Q45731350 | ||
| In utero gene transfer of human factor IX to fetal mice can induce postnatal tolerance of the exogenous clotting factor | Q45855716 | ||
| AAV vector integration sites in mouse hepatocellular carcinoma. | Q45869780 | ||
| Factors influencing in vivo transduction by recombinant adeno-associated viral vectors expressing the human factor IX cDNA. | Q45875077 | ||
| No evidence for tumorigenesis of AAV vectors in a large-scale study in mice. | Q45885410 | ||
| Expansions of transgene repeats cause heterochromatin formation and gene silencing in Drosophila. | Q52542203 | ||
| Translocations of 17q21 approximately qter in neuroblastoma cell lines infrequently include the topoisomerase IIalpha gene. | Q53623906 | ||
| P433 | issue | 5 | |
| P407 | language of work or name | English | Q1860 |
| P304 | page(s) | 876-885 | |
| P577 | publication date | 2011-01-18 | |
| P1433 | published in | Molecular Therapy | Q15762400 |
| P1476 | title | 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 | |
| P478 | volume | 19 |
| Q97423864 | A Quantitative In Vitro Potency Assay for Adeno-Associated Virus Vectors Encoding for the UGT1A1 Transgene |
| Q34994549 | A barrel of monkeys: scAAV8 gene therapy for hemophilia in nonhuman primates. |
| Q90707116 | AAV D-sequence-mediated suppression of expression of a human major histocompatibility class II gene: Implications in the development of AAV vectors for modulating humoral immune response |
| Q92876148 | AAV Vector Immunogenicity in Humans: A Long Journey to Successful Gene Transfer |
| Q42976432 | AAV vector biology in primates: finding the missing link? |
| Q35733790 | AAV-mediated gene transfer in the perinatal period results in expression of FVII at levels that protect against fatal spontaneous hemorrhage. |
| Q38981492 | Adeno-associated virus 8-mediated gene therapy for choroideremia: preclinical studies in in vitro and in vivo models |
| Q35691659 | Adenovirus-associated virus vector-mediated gene transfer in hemophilia B |
| Q37937458 | Advancements in adeno-associated viral gene therapy approaches: exploring a new horizon |
| Q59356408 | Age-Related Seroprevalence of Antibodies Against AAV-LK03 in a UK Population Cohort |
| Q36365372 | An Immune-Competent Murine Model to Study Elimination of AAV-Transduced Hepatocytes by Capsid-Specific CD8+ T Cells |
| Q35920744 | An siRNA Screen Identifies the U2 snRNP Spliceosome as a Host Restriction Factor for Recombinant Adeno-associated Viruses |
| Q35006292 | Analysis of transduction efficiency, tropism and axonal transport of AAV serotypes 1, 2, 5, 6, 8 and 9 in the mouse brain |
| Q27012571 | Animal models of hemophilia |
| Q36298627 | Balloon catheter delivery of helper-dependent adenoviral vector results in sustained, therapeutic hFIX expression in rhesus macaques |
| Q64066221 | Bile-duct proliferation as an unexpected side-effect after AAV2-LDLR gene transfer to rabbit liver |
| Q37690476 | CD8+ T cell recognition of epitopes within the capsid of adeno-associated virus 8-based gene transfer vectors depends on vectors' genome. |
| Q54941286 | CRISPR-LbCpf1 prevents choroidal neovascularization in a mouse model of age-related macular degeneration. |
| Q38754256 | Canine models of inherited bleeding disorders in the development of coagulation assays, novel protein replacement and gene therapies |
| Q35174923 | Cardiac gene transfer of short hairpin RNA directed against phospholamban effectively knocks down gene expression but causes cellular toxicity in canines |
| Q33938435 | Cell-Mediated Immunity to AAV Vectors, Evolving Concepts and Potential Solutions |
| Q26743393 | Clinical development of gene therapy: results and lessons from recent successes |
| Q42256024 | Coagulation Factor IX for Hemophilia B Therapy |
| Q59811365 | Codon-Optimization of Wild-Type Adeno-Associated Virus Capsid Sequences Enhances DNA Family Shuffling while Conserving Functionality |
| Q36563927 | Comparison of adeno-associated virus serotypes and delivery methods for cardiac gene transfer |
| Q43545330 | Comparison of gene transfer to the murine liver following intraperitoneal and intraportal delivery of hepatotropic AAV pseudo-serotypes. |
| Q39394803 | Complexity of immune responses to AAV transgene products - Example of factor IX. |
| Q92845907 | Coupling AAV-mediated promoterless gene targeting to SaCas9 nuclease to efficiently correct liver metabolic diseases |
| Q37382030 | Cre-dependent selection yields AAV variants for widespread gene transfer to the adult brain |
| Q59354563 | Cryoprecipitate augments the global transduction of the adeno-associated virus serotype 9 after a systemic administration |
| Q104289829 | Current Clinical Applications of in vivo Gene Therapy with AAVs |
| Q38206852 | Current status of haemophilia gene therapy |
| Q37132611 | Development of gene therapy for blood disorders: an update |
| Q26823376 | Developments in the treatment of hemophilia B: focus on emerging gene therapy |
| Q45325768 | Differential Prevalence of Antibodies Against Adeno-Associated Virus in Healthy Children and Patients with Mucopolysaccharidosis III: Perspective for AAV-Mediated Gene Therapy. |
| Q41935298 | Differential prevalence of antibodies against adeno-associated virus in healthy children and patients with mucopolysaccharidosis III: perspective for AAV-mediated gene therapy |
| Q43106648 | Disease correction by combined neonatal intracranial AAV and systemic lentiviral gene therapy in Sanfilippo Syndrome type B mice. |
| Q42541148 | Displaying high-affinity ligands on adeno-associated viral vectors enables tumor cell-specific and safe gene transfer |
| Q59358394 | Double-stranded RNA innate immune response activation from long-term adeno-associated virus vector transduction |
| Q38874937 | Effective Depletion of Pre-existing Anti-AAV Antibodies Requires Broad Immune Targeting |
| Q36571141 | Effects of FVIII immunity on hepatocyte and hematopoietic stem cell-directed gene therapy of murine hemophilia A. |
| Q47554736 | Emerging Issues in AAV-Mediated In Vivo Gene Therapy |
| Q37950457 | Endogenous migration modulators as parent compounds for the development of novel cardiovascular and anti-inflammatory drugs |
| Q36709834 | Engineered AAV vector minimizes in vivo targeting of transduced hepatocytes by capsid-specific CD8+ T cells. |
| Q36951529 | Enhanced T cell function in a mouse model of human glycosylation. |
| Q38890789 | Extracting structural and functional features of widely distributed biological circuits with single cell resolution via tissue clearing and delivery vectors |
| Q37881971 | Fetal gene therapy: recent advances and current challenges |
| Q91938979 | Five Years of Successful Inducible Transgene Expression Following Locoregional Adeno-Associated Virus Delivery in Nonhuman Primates with No Detectable Immunity |
| Q59355726 | Gene Therapy for C1 Esterase Inhibitor Deficiency in a Murine Model of Hereditary Angioedema |
| Q28595868 | Gene Therapy for Metabolic Diseases |
| Q40103301 | Gene Therapy in Tyrosinemia: Potential and Pitfalls. |
| Q45338599 | Gene Therapy with Adeno-associated Virus for Cystic Fibrosis |
| Q47225946 | Gene Therapy with BMN 270 Results in Therapeutic Levels of FVIII in Mice and Primates and Normalization of Bleeding in Hemophilic Mice |
| Q45879900 | Gene therapy for haemophilia B. |
| Q35670173 | Gene therapy for haemophilia: prospects and challenges to prevent or reverse inhibitor formation |
| Q28081911 | Gene therapy for hemophilia |
| Q40814418 | Gene therapy for hemophilia B mice with scAAV8-LP1-hFIX. |
| Q38803022 | Gene therapy for immune tolerance induction in hemophilia with inhibitors |
| Q33881920 | Gene therapy for monogenic liver diseases: clinical successes, current challenges and future prospects. |
| Q36634890 | Gene therapy for mucopolysaccharidosis type VI is effective in cats without pre-existing immunity to AAV8. |
| Q57165501 | Gene therapy for neurological disorders: progress and prospects |
| Q38076953 | Hemophilia clinical gene therapy: brief review |
| Q45883899 | Hepatic lentiviral gene transfer is associated with clonal selection, but not with tumor formation in serially transplanted rodents. |
| Q41575309 | High cerebrospinal fluid levels of interleukin-10 attained by AAV in dogs |
| Q90196148 | Homologous Recombination-Based Genome Editing by Clade F AAVs Is Inefficient in the Absence of a Targeted DNA Break |
| Q94502776 | Human Immune Responses to Adeno-Associated Virus (AAV) Vectors |
| Q96127660 | IgG-cleaving endopeptidase enables in vivo gene therapy in the presence of anti-AAV neutralizing antibodies |
| Q90000829 | Immunoadsorption enables successful rAAV5-mediated repeated hepatic gene delivery in nonhuman primates |
| Q40219232 | In Utero Transfer of Adeno-Associated Viral Vectors Produces Long-Term Factor IX Levels in a Cynomolgus Macaque Model. |
| Q36264921 | Induction of Immune Tolerance to Foreign Protein via Adeno-Associated Viral Vector Gene Transfer in Mid-Gestation Fetal Sheep |
| Q36094151 | Induction of hepatocellular carcinoma by in vivo gene targeting |
| Q54210526 | Influence of Pre-existing Anti-capsid Neutralizing and Binding Antibodies on AAV Vector Transduction. |
| Q34380850 | Inhibitors - cellular aspects and novel approaches for tolerance |
| Q37939843 | Innate Immune Responses to AAV Vectors |
| Q39130497 | Insight into the mechanisms of enhanced retinal transduction by the engineered AAV2 capsid variant -7m8. |
| Q36014088 | Integration frequency and intermolecular recombination of rAAV vectors in non-human primate skeletal muscle and liver |
| Q35821871 | Intravenous AAV8 Encoding Urocortin-2 Increases Function of the Failing Heart in Mice |
| Q33586309 | Limitations of encapsidation of recombinant self-complementary adeno-associated viral genomes in different serotype capsids and their quantitation |
| Q88166600 | Liver induced transgene tolerance with AAV vectors |
| Q39553052 | Liver-Directed Adeno-Associated Viral Gene Therapy for Hemophilia |
| Q41571631 | Long-term glycemic control with hepatic insulin gene therapy in streptozotocin-diabetic mice |
| Q34783922 | Long-term safety and efficacy of factor IX gene therapy in hemophilia B. |
| Q37507816 | Low-dose Gene Therapy Reduces the Frequency of Enzyme Replacement Therapy in a Mouse Model of Lysosomal Storage Disease |
| Q26750799 | Manufacturing of recombinant adeno-associated viral vectors for clinical trials |
| Q89956643 | Membrane fusion FerA domains enhance adeno-associated virus vector transduction |
| 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 |
| Q34470424 | Modeling Pulmonary Disease Pathways Using Recombinant Adeno-Associated Virus 6.2. |
| Q90605117 | Molecular Engineering of Adeno-Associated Virus Capsid Improves Its Therapeutic Gene Transfer in Murine Models of Hemophilia and Retinal Degeneration |
| Q92940638 | Muscle-Directed Delivery of an AAV1 Vector Leads to Capsid-Specific T Cell Exhaustion in Nonhuman Primates and Humans |
| Q36825929 | Neutralizing Antibodies Against Adeno-Associated Viral Capsids in Patients with mut Methylmalonic Acidemia |
| Q38622440 | New and improved AAVenues: current status of hemophilia B gene therapy |
| Q41347222 | Non-clinical Safety and Efficacy of an AAV2/8 Vector Administered Intravenously for Treatment of Mucopolysaccharidosis Type VI. |
| Q38562869 | Our journey to successful gene therapy for hemophilia B. |
| Q42056009 | Overcoming preexisting humoral immunity to AAV using capsid decoys |
| Q35238390 | Perinatal gene transfer to the liver |
| Q40709550 | Pharmacodynamics of anti-HIV gene therapy using viral vectors and targeted endonucleases. |
| Q36085401 | Pharmacological modulation of humoral immunity in a nonhuman primate model of AAV gene transfer for hemophilia B. |
| Q38012709 | Phoenix rising: gene therapy makes a comeback |
| Q37993976 | Platelet and endothelial expression of clotting factors for the treatment of hemophilia |
| Q37690441 | Platelet gene therapy by lentiviral gene delivery to hematopoietic stem cells restores hemostasis and induces humoral immune tolerance in FIX(null) mice |
| Q34795284 | Portal vein delivery of viral vectors for gene therapy for hemophilia |
| Q35673189 | Preclinical Potency and Biodistribution Studies of an AAV 5 Vector Expressing Human Interferon-β (ART-I02) for Local Treatment of Patients with Rheumatoid Arthritis |
| Q35750005 | Preclinical dose-finding study with a liver-tropic, recombinant AAV-2/8 vector in the mouse model of galactosialidosis |
| Q42175418 | Preferential targeting of disseminated liver tumors using a recombinant adeno-associated viral vector. |
| Q90012812 | Prevalence and Relevance of Pre-Existing Anti-Adeno-Associated Virus Immunity in the Context of Gene Therapy for Crigler-Najjar Syndrome |
| 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. |
| Q34433586 | Prevention of muscular dystrophy in mice by CRISPR/Cas9-mediated editing of germline DNA |
| Q92487813 | Progress and challenges of gene therapy for Pompe disease |
| Q38601073 | Progress toward improved therapies for inborn errors of metabolism |
| Q33844184 | Progress towards gene therapy for haemophilia B. |
| Q28730577 | Proteasome inhibition is partially effective in attenuating pre-existing immunity against recombinant adeno-associated viral vectors |
| 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 |
| 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 |
| 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 |
| Q38716558 | Sodium taurocholate cotransporting polypeptide is the limiting host factor of hepatitis B virus infection in macaque and pig hepatocytes |
| Q35568499 | Stable human FIX expression after 0.9G intrauterine gene transfer of self-complementary adeno-associated viral vector 5 and 8 in macaques |
| Q45874009 | Strategy to detect pre-existing immunity to AAV gene therapy. |
| Q35969194 | Successful Phenotype Improvement following Gene Therapy for Severe Hemophilia A in Privately Owned Dogs. |
| Q40166892 | Successful Repeated Hepatic Gene Delivery in Mice and Non-human Primates Achieved by Sequential Administration of AAV5ch and AAV1. |
| Q37043507 | Superior In vivo Transduction of Human Hepatocytes Using Engineered AAV3 Capsid |
| Q90390816 | Superior human hepatocyte transduction with adeno-associated virus vector serotype 7 |
| Q36013060 | Synergistic effects of central nervous system-directed gene therapy and bone marrow transplantation in the murine model of infantile neuronal ceroid lipofuscinosis |
| Q36807045 | Systemic errors in quantitative polymerase chain reaction titration of self-complementary adeno-associated viral vectors and improved alternative methods |
| Q52649447 | Target-Cell-Directed Bioengineering Approaches for Gene Therapy of Hemophilia A. |
| Q64039743 | The Alpha-1-Antitrypsin Promoter Improves the Efficacy of an AAV Vector for the Treatment of MNGIE |
| Q64056230 | The Impact of Pre-existing Immunity on the Non-clinical Pharmacodynamics of AAV5-Based Gene Therapy |
| Q26751281 | The interplay of post-translational modification and gene therapy |
| Q42261520 | The prevalence of neutralizing antibodies against adeno-associated virus capsids is reduced in young Japanese individuals |
| Q36155013 | The threefold protrusions of adeno-associated virus type 8 are involved in cell surface targeting as well as postattachment processing |
| Q90275396 | Therapeutic expression of human clotting factors IX and X following adeno-associated viral vector-mediated intrauterine gene transfer in early-gestation fetal macaques |
| Q91645665 | Therapeutic hFIX Activity Achieved after Single AAV5-hFIX Treatment in Hemophilia B Patients and NHPs with Pre-existing Anti-AAV5 NABs |
| Q36797248 | Therapeutic levels of FVIII following a single peripheral vein administration of rAAV vector encoding a novel human factor VIII variant |
| Q64100888 | Threshold for Pre-existing Antibody Levels Limiting Transduction Efficiency of Systemic rAAV9 Gene Delivery: Relevance for Translation |
| Q96109656 | Timing of Intensive Immunosuppression Impacts Risk of Transgene Antibodies after AAV Gene Therapy in Nonhuman Primates |
| Q41532325 | Transient suppression of hepatocellular replication in the mouse liver following transduction with recombinant adeno-associated virus. |
| Q27335825 | Ultramicroscopy as a novel tool to unravel the tropism of AAV gene therapy vectors in the brain |
| Q91059831 | Urocortin 2 Gene Transfer Improves Heart Function in Aged Mice |
| Q88985852 | Wang et al. reply |
| Q30541880 | Whole body correction of mucopolysaccharidosis IIIA by intracerebrospinal fluid gene therapy |
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