期刊
MOLECULAR THERAPY-METHODS & CLINICAL DEVELOPMENT
卷 12, 期 -, 页码 248-265出版社
CELL PRESS
DOI: 10.1016/j.omtm.2019.01.008
关键词
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资金
- Federal Ministry of Education and Research (BMBF)
- Ministry for Science and Culture of Lower Saxony (MWK)
- German Research Foundation (DFG)
- NIH [R01 HL-097088, R41 AI-122735, R21 EB-015684]
- Children's Miracle Network
- Kitzman Foundation
In the past decade, recombinant vectors based on a non-pathogenic parvovirus, the adeno-associated virus (AAV), have taken center stage as a gene delivery vehicle for the potential gene therapy for a number of human diseases. To date, the safety of AAV vectors in 176 phase I, II, and III clinical trials and their efficacy in at least eight human diseases are now firmly documented. Despite these remarkable achievements, it has also become abundantly clear that the full potential of first generation AAV vectors composed of naturally occurring capsids is not likely to be realized, since the wild-type AAV did not evolve for the purpose of therapeutic gene delivery. In this article, we provide a brief historical account of the progress that has been made in the development of capsid-modified, next-generation AAV vectors to ensure both the safety and efficacy of these vectors in targeting a wide variety of human diseases.
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