Gene Therapy for Mucopolysaccharidosis Type VI Is Effective in Cats Without Pre-Existing Immunity to AAV8
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Title
Gene Therapy for Mucopolysaccharidosis Type VI Is Effective in Cats Without Pre-Existing Immunity to AAV8
Authors
Keywords
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Journal
HUMAN GENE THERAPY
Volume 24, Issue 2, Pages 163-169
Publisher
Mary Ann Liebert Inc
Online
2012-11-30
DOI
10.1089/hum.2012.179
References
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Related references
Note: Only part of the references are listed.- Impact of Age at Administration, Lysosomal Storage, and Transgene Regulatory Elements on AAV2/8-Mediated Rat Liver Transduction
- (2012) Gabriella Cotugno et al. PLoS One
- Impact of Pre-Existing Immunity on Gene Transfer to Nonhuman Primate Liver with Adeno-Associated Virus 8 Vectors
- (2011) Lili Wang et al. HUMAN GENE THERAPY
- 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
- (2011) Amit C Nathwani et al. MOLECULAR THERAPY
- Adenovirus-Associated Virus Vector–Mediated Gene Transfer in Hemophilia B
- (2011) Amit C. Nathwani et al. NEW ENGLAND JOURNAL OF MEDICINE
- 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
- (2010) Sylvie Boutin et al. HUMAN GENE THERAPY
- Preexisting Immunity and Low Expression in Primates Highlight Translational Challenges for Liver-directed AAV8-mediated Gene Therapy
- (2010) Gregory D Hurlbut et al. MOLECULAR THERAPY
- Long-term Amelioration of Feline Mucopolysaccharidosis VI After AAV-mediated Liver Gene Transfer
- (2010) Gabriella Cotugno et al. MOLECULAR THERAPY
- Effect of Adeno-Associated Virus Serotype and Genomic Structure on Liver Transduction and Biodistribution in Mice of Both Genders
- (2009) Astrid Pañeda et al. HUMAN GENE THERAPY
- Different Serum Enzyme Levels Are Required to Rescue the Various Systemic Features of the Mucopolysaccharidoses
- (2009) Gabriella Cotugno et al. HUMAN GENE THERAPY
- Worldwide Epidemiology of Neutralizing Antibodies to Adeno‐Associated Viruses
- (2009) Roberto Calcedo et al. JOURNAL OF INFECTIOUS DISEASES
- The Pleiotropic Effects of Natural AAV Infections on Liver-directed Gene Transfer in Macaques
- (2009) Lili Wang et al. MOLECULAR THERAPY
- Systematic Evaluation of AAV Vectors for Liver directed Gene Transfer in Murine Models
- (2009) Lili Wang et al. MOLECULAR THERAPY
- Enhancing transduction of the liver by adeno-associated viral vectors
- (2008) A C Nathwani et al. GENE THERAPY
- Long-term follow-up of endurance and safety outcomes during enzyme replacement therapy for mucopolysaccharidosis VI: Final results of three clinical studies of recombinant human N-acetylgalactosamine 4-sulfatase
- (2008) Paul Harmatz et al. MOLECULAR GENETICS AND METABOLISM
- Biochemical, Pathological, and Skeletal Improvement of Mucopolysaccharidosis VI After Gene Transfer to Liver but Not to Muscle
- (2007) Alessandra Tessitore et al. MOLECULAR THERAPY
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