期刊
GASTROENTEROLOGY CLINICS OF NORTH AMERICA
卷 48, 期 2, 页码 319-+出版社
W B SAUNDERS CO-ELSEVIER INC
DOI: 10.1016/j.gtc.2019.02.005
关键词
AAV vectors; Gene transfer; Human liver diseases; Gene therapy
资金
- Public Health Service grants from the National Institutes of Health [R01 HL-097088, R41 AI-122735, R21 EB-015684]
- Children's Miracle Network
- Kitzman Foundation
Recombinant vectors based on a nonpathogenic parvovirus, the adeno-associated virus (AAV), have taken center stage in the past decade. The safety of AAV vectors in clinical trials and clinical efficacy in several human diseases are now well documented. Despite these achievements, it is increasingly clear that the full potential of AAV vectors composed of the naturally occurring capsids is unlikely to be realized. This article describes advances that have been made and challenges that remain in the optimal use of MV vectors in human gene therapy applications.
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