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Neonatal gene therapy achieves sustained disease rescue of maple syrup urine disease in mice.
- Published in:
- Nature Communications, 2022, v. 13, n. 1, p. 1, doi. 10.1038/s41467-022-30880-w
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- Publication type:
- Article
Neonatal gene therapy achieves sustained disease rescue of maple syrup urine disease in mice.
- Published in:
- Nature Communications, 2022, v. 13, n. 1, p. 1, doi. 10.1038/s41467-022-30880-w
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- Publication type:
- Article
Neonatal gene therapy achieves sustained disease rescue of maple syrup urine disease in mice.
- Published in:
- Nature Communications, 2022, v. 13, n. 1, p. 1, doi. 10.1038/s41467-022-30880-w
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- Publication type:
- Article
Single‐domain antibodies targeting antithrombin reduce bleeding in hemophilic mice with or without inhibitors.
- Published in:
- EMBO Molecular Medicine, 2020, v. 12, n. 4, p. 1, doi. 10.15252/emmm.201911298
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- Publication type:
- Article
Whole body correction of mucopolysaccharidosis IIIA by intracerebrospinal fluid gene therapy.
- Published in:
- 2013
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- Publication type:
- journal article
Capsid antigen presentation flags human hepatocytes for destruction after transduction by adeno-associated viral vectors.
- Published in:
- 2009
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- Publication type:
- journal article
Diverse IgG subclass responses to adeno-associated virus infection and vector administration.
- Published in:
- Journal of Medical Virology, 2009, v. 81, n. 1, p. 65, doi. 10.1002/jmv.21360
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- Publication type:
- Article
Emerging Immunogenicity and Genotoxicity Considerations of Adeno-Associated Virus Vector Gene Therapy for Hemophilia.
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- Journal of Clinical Medicine, 2021, v. 10, n. 11, p. 2471, doi. 10.3390/jcm10112471
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- Publication type:
- Article
Pre-existing humoral immunity and complement pathway contribute to immunogenicity of adeno-associated virus (AAV) vector in human blood.
- Published in:
- Frontiers in Immunology, 2022, v. 13, p. 1, doi. 10.3389/fimmu.2022.999021
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- Publication type:
- Article
Gateway to the diseased brain.
- Published in:
- Nature Medicine, 2009, v. 15, n. 10, p. 1123, doi. 10.1038/nm1009-1123
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- Publication type:
- Article
CD8<sup>+</sup> T-cell responses to adeno-associated virus capsid in humans.
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- Nature Medicine, 2007, v. 13, n. 4, p. 419, doi. 10.1038/nm1549
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- Publication type:
- Article
Long-term exposure to Myozyme results in a decrease of anti-drug antibodies in late-onset Pompe disease patients.
- Published in:
- Scientific Reports, 2016, p. 36182, doi. 10.1038/srep36182
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- Publication type:
- Article
Preclinical Potency and Biodistribution Studies of an AAV 5 Vector Expressing Human Interferon-β (ART-I02) for Local Treatment of Patients with Rheumatoid Arthritis.
- Published in:
- PLoS ONE, 2015, v. 10, n. 6, p. 1, doi. 10.1371/journal.pone.0130612
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- Publication type:
- Article
Th1 Response and Systemic Treg Deficiency in Inclusion Body Myositis.
- Published in:
- PLoS ONE, 2014, v. 9, n. 3, p. 1, doi. 10.1371/journal.pone.0088788
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- Publication type:
- Article
IL12-Mediated Liver Inflammation Reduces the Formation of AAV Transcriptionally Active Forms but Has No Effect over Preexisting AAV Transgene Expression.
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- PLoS ONE, 2013, v. 8, n. 7, p. 1, doi. 10.1371/journal.pone.0067748
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- Publication type:
- Article
Deep characterization of the anti-drug antibodies developed in Fabry disease patients, a prospective analysis from the French multicenter cohort FFABRY.
- Published in:
- 2018
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- Publication type:
- journal article
Therapeutic in vivo gene transfer for genetic disease using AAV: progress and challenges.
- Published in:
- 2011
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- Publication type:
- Correction Notice
Therapeutic in vivo gene transfer for genetic disease using AAV: progress and challenges.
- Published in:
- 2011
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- Publication type:
- journal article
Deep morphological analysis of muscle biopsies from type III glycogenesis (GSDIII), debranching enzyme deficiency, revealed stereotyped vacuolar myopathy and autophagy impairment.
- Published in:
- Acta Neuropathologica Communications, 2019, v. 7, n. 1, p. N.PAG, doi. 10.1186/s40478-019-0815-2
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- Publication type:
- Article
Human Immune Responses to Adeno-Associated Virus (AAV) Vectors.
- Published in:
- Frontiers in Immunology, 2020, v. 11, p. 1, doi. 10.3389/fimmu.2020.00670
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- Publication type:
- Article
Disease burden and management of Crigler‐Najjar syndrome: Report of a world registry.
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- Liver International, 2022, v. 42, n. 7, p. 1593, doi. 10.1111/liv.15239
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- Publication type:
- Article
Modulation of CD8<sup>+</sup> T cell responses to AAV vectors with IgG-derived MHC class II epitopes.
- Published in:
- Molecular Therapy, 2013, v. 21, n. 9, p. 1, doi. 10.1038/mt.2013.166
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- Publication type:
- Article
Pharmacological Modulation of Humoral Immunity in a Nonhuman Primate Model of AAV Gene Transfer for Hemophilia B.
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- Molecular Therapy, 2012, v. 20, n. 7, p. 1410, doi. 10.1038/mt.2012.84
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- Publication type:
- Article
In vivo engineering of lymphocytes after systemic exosome-associated AAV delivery.
- Published in:
- Scientific Reports, 2020, v. 10, n. 1, p. 1, doi. 10.1038/s41598-020-61518-w
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- Publication type:
- Article
Exposure to wild-type AAV drives distinct capsid immunity profiles in humans.
- Published in:
- 2018
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- Publication type:
- journal article
In Vitro and In Vivo Studies of IgG-derived Treg Epitopes (Tregitopes): A Promising New Tool for Tolerance Induction and Treatment of Autoimmunity.
- Published in:
- Journal of Clinical Immunology, 2013, v. 33, n. 1, p. 43, doi. 10.1007/s10875-012-9762-4
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- Publication type:
- Article
Successful treatment of severe MSUD in Bckdhb<sup>−/−</sup> mice with neonatal AAV gene therapy.
- Published in:
- Journal of Inherited Metabolic Disease, 2024, v. 47, n. 1, p. 41, doi. 10.1002/jimd.12604
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- Publication type:
- Article
Induction of Hematopoietic Microchimerism by Gene-Modified BMT Elicits Antigen-Specific B and T Cell Unresponsiveness toward Gene Therapy Products.
- Published in:
- Frontiers in Immunology, 2016, p. 1, doi. 10.3389/fimmu.2016.00360
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- Publication type:
- Article
Exon-specific U1 snRNAs improve ELP1 exon 20 definition and rescue ELP1 protein expression in a familial dysautonomia mouse model.
- Published in:
- Human Molecular Genetics, 2018, v. 27, n. 14, p. 2466, doi. 10.1093/hmg/ddy151
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- Publication type:
- Article
Biochemical, histological and functional correction of mucopolysaccharidosis Type IIIB by intra-cerebrospinal fluid gene therapy.
- Published in:
- Human Molecular Genetics, 2015, v. 24, n. 7, p. 2078, doi. 10.1093/hmg/ddu727
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- Publication type:
- Article
Overcoming Preexisting Humoral Immunity to AAV Using Capsid Decoys.
- Published in:
- Science Translational Medicine, 2013, v. 5, n. 194, p. 1, doi. 10.1126/scitranslmed.3005795
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- Publication type:
- Article
AAV2 Gene Therapy Readministration in Three Adults with Congenital Blindness.
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- Science Translational Medicine, 2012, v. 4, n. 120, p. 1, doi. 10.1126/scitranslmed.3002865
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- Publication type:
- Article
Safety and Efficacy of Subretinal Readministration of a Viral Vector in Large Animals to Treat Congenital Blindness.
- Published in:
- Science Translational Medicine, 2010, v. 2, n. 21, p. 1, doi. 10.1126/scitranslmed.3000659
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- Publication type:
- Article
Adeno-Associated Virus Vector-Based Gene Therapy for Monogenetic Metabolic Diseases of the Liver.
- Published in:
- Journal of Pediatric Gastroenterology & Nutrition, 2015, v. 60, n. 4, p. 433, doi. 10.1097/MPG.0000000000000703
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- Publication type:
- Article
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.
- Published in:
- Molecular Therapy, 2011, v. 19, n. 5, p. 876, doi. 10.1038/mt.2010.274
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- Publication type:
- Article
Safety of AAV Factor IX Peripheral Transvenular Gene Delivery to Muscle in Hemophilia B Dogs.
- Published in:
- Molecular Therapy, 2010, v. 18, n. 7, p. 1318, doi. 10.1038/mt.2010.73
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- Publication type:
- Article
Gene Therapy for Leber's Congenital Amaurosis is Safe and Effective Through 1.5 Years After Vector Administration.
- Published in:
- Molecular Therapy, 2010, v. 18, n. 3, p. 643, doi. 10.1038/mt.2009.277
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- Publication type:
- Article
Proteasome Inhibitors Decrease AAV2 Capsid derived Peptide Epitope Presentation on MHC Class I Following Transduction.
- Published in:
- Molecular Therapy, 2010, v. 18, n. 1, p. 135, doi. 10.1038/mt.2009.257
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- Publication type:
- Article
Host and Vector-dependent Effects on the Risk of Germline Transmission of AAV Vectors.
- Published in:
- Molecular Therapy, 2009, v. 17, n. 6, p. 1022, doi. 10.1038/mt.2009.56
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- Publication type:
- Article
Undetectable Transcription of cap in a Clinical AAV Vector: Implications for Preformed Capsid in Immune Responses.
- Published in:
- Molecular Therapy, 2009, v. 17, n. 1, p. 144, doi. 10.1038/mt.2008.227
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- Publication type:
- Article
Reversal of Blindness in Animal Models of Leber Congenital Amaurosis Using Optimized AAV2-mediated Gene Transfer.
- Published in:
- Molecular Therapy, 2008, v. 16, n. 3, p. 458, doi. 10.1038/sj.mt.6300389
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- Publication type:
- Article
1086. T Cell Responses to Canine Factor IX and AAV Capsid Antigens in Hemophilia B Dogs after Intravascular Gene Delivery to Skeletal Muscle.
- Published in:
- Molecular Therapy, 2006, v. 13, p. S417, doi. 10.1016/j.ymthe.2006.08.1188
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- Publication type:
- Article
74. Cellular Immunity to Adeno-Associated Virus Capsid Attenuates Transgene Expression in the Liver.
- Published in:
- Molecular Therapy, 2006, v. 13, p. S31, doi. 10.1016/j.ymthe.2006.08.091
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- Publication type:
- Article
75. Protease-Activated Receptor 2 as a Novel Target for Immune Modulation for AAV-Mediated Gene Transfer to Skeletal Muscle.
- Published in:
- Molecular Therapy, 2006, v. 13, p. S32, doi. 10.1016/j.ymthe.2006.08.092
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- Publication type:
- Article
110. Investigation of Biochemical Factors That May Influence Immunogencity of AAV2 Vectors.
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- Molecular Therapy, 2006, v. 13, p. S45, doi. 10.1016/j.ymthe.2006.08.130
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- Publication type:
- Article
730. T Cell Responses to AAV Vector Capsid in Normal Donors and Subjects Who Have Undergone Liver-Directed AAV-Mediated Gene Transfer.
- Published in:
- Molecular Therapy, 2006, v. 13, p. S282, doi. 10.1016/j.ymthe.2006.08.810
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- Publication type:
- Article
Identification of mouse AAV capsid-specific CD8+ T cell epitopes
- Published in:
- Molecular Therapy, 2005, v. 12, n. 6, p. 1023, doi. 10.1016/j.ymthe.2005.09.009
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- Publication type:
- Article
609. Long Term Persistence of Canine Factor IX-Specific Th1 Lymphocytes in Regional Lymph Nodes after AAV1-Mediated Intramuscular Gene Transfer in a Hemophilia B Dog
- Published in:
- 2005
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- Publication type:
- Abstract
614. Frequency of T Cell Responses to AAV-2 Capsid in Peripheral Blood Mononuclear Cells of Normal Subjects Does Not Correlate with Anti-AAV2 Antibody Titers
- Published in:
- 2005
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- Publication type:
- Abstract
132. An Optimized CsCl Gradient -Based Method to Generate High Concentration AAV Vector Stocks of High Purity and In Vivo Potency
- Published in:
- 2005
- By:
- Publication type:
- Abstract