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Title
Development of Gene Therapy Vectors: Remaining Challenges
Authors
Keywords
Adeno-associated virus (AAV), Gene delivery, Gene therapy, Neutralizing antibody (Nab) assay, Potency assay
Journal
JOURNAL OF PHARMACEUTICAL SCIENCES
Volume -, Issue -, Pages -
Publisher
Elsevier BV
Online
2020-12-04
DOI
10.1016/j.xphs.2020.11.035
References
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- (2019) Anita Kruzik et al. Human Gene Therapy Methods
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- (2019) Dan Wang et al. NATURE REVIEWS DRUG DISCOVERY
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- (2019) Fazhan Wang et al. JOURNAL OF GENE MEDICINE
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- Emerging Issues in AAV-Mediated In Vivo Gene Therapy
- (2018) Pasqualina Colella et al. Molecular Therapy-Methods & Clinical Development
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- Comparative analyses of adeno-associated viral vector serotypes 1, 2, 5, 8 and 9 in marmoset, mouse and macaque cerebral cortex
- (2015) Akiya Watakabe et al. NEUROSCIENCE RESEARCH
- Adeno-Associated Virus 9-Mediated Airway Expression of Antibody Protects Old and Immunodeficient Mice against Influenza Virus
- (2014) Virginie S. Adam et al. Clinical and Vaccine Immunology
- AAV Empty Capsids: For Better or for Worse?
- (2014) J Fraser Wright MOLECULAR THERAPY
- Long-Term Safety and Efficacy of Factor IX Gene Therapy in Hemophilia B
- (2014) Amit C. Nathwani et al. NEW ENGLAND JOURNAL OF MEDICINE
- Immune responses to AAV vectors: overcoming barriers to successful gene therapy
- (2013) F. Mingozzi et al. BLOOD
- Vectored Expression of the Broadly Neutralizing Antibody FI6 in Mouse Airway Provides Partial Protection against a New Avian Influenza A Virus, H7N9
- (2013) Maria P. Limberis et al. Clinical and Vaccine Immunology
- Overcoming Preexisting Humoral Immunity to AAV Using Capsid Decoys
- (2013) F. Mingozzi et al. Science Translational Medicine
- Advances in Liver-Directed Gene Therapy for Hepatocellular Carcinoma by Non-Viral Delivery Systems
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- (2012) F Mingozzi et al. GENE THERAPY
- Development and validation of cell-based assays for the detection of neutralizing antibodies to drug products: a practical approach
- (2012) Pierre Jolicoeur et al. Bioanalysis
- The genome of self-complementary adeno-associated viral vectors increases Toll-like receptor 9-dependent innate immune responses in the liver
- (2011) A. T. Martino et al. BLOOD
- Adeno-Associated Virus Antibody Profiles in Newborns, Children, and Adolescents
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- Neutralizing antibodies against adeno-associated virus examined prospectively in pediatric patients with hemophilia
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- Impact of Pre-Existing Immunity on Gene Transfer to Nonhuman Primate Liver with Adeno-Associated Virus 8 Vectors
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- Systemic Administration of AAV8-α-galactosidase A Induces Humoral Tolerance in Nonhuman Primates Despite Low Hepatic Expression
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- Adenovirus-Associated Virus Vector–Mediated Gene Transfer in Hemophilia B
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- Efficient Serotype-Dependent Release of Functional Vector into the Culture Medium During Adeno-Associated Virus Manufacturing
- (2010) Luk H. Vandenberghe et al. HUMAN GENE THERAPY
- Prevalence of Serum IgG and Neutralizing Factors Against Adeno-Associated Virus (AAV) Types 1, 2, 5, 6, 8, and 9 in the Healthy Population: Implications for Gene Therapy Using AAV Vectors
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