Feasibility and Safety of Systemic rAAV9-hNAGLU Delivery for Treating Mucopolysaccharidosis IIIB: Toxicology, Biodistribution, and Immunological Assessments in Primates-Reference-Cited by-同舟云学术

Feasibility and Safety of Systemic rAAV9-hNAGLU Delivery for Treating Mucopolysaccharidosis IIIB: Toxicology, Biodistribution, and Immunological Assessments in Primates

Published:2014-06 Issue:2 Volume:25 Page:72-84
ISSN:2324-8637
Container-title:Human Gene Therapy Clinical Development
language:en
Short-container-title:Human Gene Therapy Clinical Development

Author:

Murrey Darren A.¹,Naughton Bartholomew J.¹,Duncan F. Jason¹,Meadows Aaron S.¹,Ware Tierra A.¹,Campbell Katie J.²,Bremer William G.²,Walker Christopher M.²³,Goodchild Laurie⁴,Bolon Brad⁵,La Perle Krista⁵,Flanigan Kevin M.¹³,McBride Kim L.³⁶,McCarty Douglas M.¹³,Fu Haiyan¹³

Affiliation:

1. Center for Gene Therapy, The Research Institute at Nationwide Children's Hospital, Columbus, OH 43205.

2. Center for Vaccines and Immunity, The Research Institute at Nationwide Children's Hospital, Columbus, OH 43205.

3. Department of Pediatrics, College of Medicine and Public Health, The Ohio State University, Columbus, OH 43210.

4. Vivarium, The Research Institute at Nationwide Children's Hospital, Columbus, OH 43205.

5. Comparative Pathology and Mouse Phenotyping Shared Resource, Comprehensive Cancer Center, College of Medicine and Public Health, The Ohio State University, Columbus, OH 43210.

6. Center for Cardiovascular and Pulmonary Research, The Research Institute at Nationwide Children's Hospital, Columbus, OH 43205.

Publisher

Mary Ann Liebert Inc

Subject

Genetics(clinical)

Link

http://www.liebertpub.com/doi/pdf/10.1089/humc.2013.208

Reference48 articles.

1. AAV-Mediated Gene Delivery in Adult GM1-Gangliosidosis Mice Corrects Lysosomal Storage in CNS and Improves Survival

2. The cryptic life style of adenoassociated virus

3. Systemic Gene Delivery in Large Species for Targeting Spinal Cord, Brain, and Peripheral Tissues for Pediatric Disorders

4. 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

5. Gene Therapy Using Adeno-Associated Virus Vectors

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