Systematic Evaluation of AAV Vectors for Liver directed Gene Transfer in Murine Models |
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| Authors: | Lili Wang Huan Wang Peter Bell Robert J McCarter Jianping He Roberto Calcedo Luk H Vandenberghe Hiroki Morizono Mark L Batshaw James M Wilson |
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| Affiliation: | 1Gene Therapy Program, Department of Pathology and Laboratory Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, USA;2Vaccine Research Institute, Third Affiliated Hospital, Sun Yat-Sen University, Guangzhou, China;3Children''s National Medical Center, Department of Pediatrics, George Washington University School of Medicine and Health Sciences, Washington, DC, USA |
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| Abstract: | Vectors based on adeno-associated viruses (AAVs) are being evaluated for use in liver-directed gene therapy. Candidates have been preselected on the basis of capsid structure that plays an important role in determining performance profiles. We describe a comprehensive and statistically powered set of mouse studies designed to compare the performance of vectors based on seven novel AAV capsids. The key criteria used to select candidates for successful gene therapy are high level and stable transgene expression in the absence of toxicity. Based on these criteria, the best performing vectors, AAV8, AAVhu.37, and AAVrh.8, will be further evaluated in nonhuman primates (NHPs). |
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