Adeno-associated virus (AAV) vectors are the leading platform for gene delivery for the treatment of a variety of human diseases. Recent advances in developing clinically desirable AAV capsids, optimizing genome designs and harnessing revolutionary biote
Adeno-associated virus (AAV) vectors are the leading platform for gene delivery for the treatment of a variety of human diseases. Recent advances in developing clinically desirable AAV capsids, optimizing genome designs and harnessing revolutionary biotechnologies have contributed substantially to the ...
Adeno-associated virus vector as a platform for gene therapy delivery. Nat. Rev. Drug Discov. 18, 358–378 (2019). Article CAS PubMed PubMed Central Google Scholar Ylä-Herttuala, S. Endgame: glybera finally recommended for approval as the first gene therapy drug in the European union....
Adeno-associated virus (AAV) vectors exhibit a number of properties that have made this vector system an excellent choice for both CNS gene therapy and bas... J Mccown,Thomas - 《Current Gene Therapy》 被引量: 209发表: 2011年 Adeno-associated virus of a single-polarity DNA genome is capabl...
Wang D, Tai PWL, Gao G (2019) Adeno-associated virus vector as a platform for gene therapy delivery.Nat Rev Drug Discov18(5):358–378. ClinicalTrials.gov. U.S. National Library of Medicine. Patheon by Thermo Fisher Scientific. Viral Vector Services. https://patheon.com/viral-vect...
【答案】：AAV是一类单链线状DNA缺陷型病毒。其基因组DNA小于5kh，无包膜。AAV不能独立复制，只有在辅助病毒存在时，才能进行复制和溶细胞性感染，否则只能建立溶源性潜伏感染。AAV能高效定点整合至人染色体中，避免随机整合可能带来的抑癌基因失活和原癌基因激活的潜在危险性。
and this can limit effective gene transfer and nullify transgene expression. Several approaches to AAV vector engineering offer solutions to its limitations and also improve its potential as a gene delivery platform; for example, the inverted terminal repeats (ITRs) can be modified ...
Recombinant adeno-associated virus (AAV) vectors have been used to transduce murine skeletal muscle as a platform for secretion of therapeutic proteins. Th... S Song,M Morgan,T Ellis,... - 《Proceedings of the National Academy of Sciences》 被引量: 645发表: 1998年 Binding of adeno-associate...
Adeno-associated virus (AAV) has become an increasingly important platform vector for in vivo gene therapy due to its safety profile [1,2]. In contrast to other viral vectors, it does not cause any known diseases in humans, has a lower immunogenicity, primarily expresses episomally, and has...
Adeno-associated virus (AAV) is a promising platform for delivering a transgene that has low pathogenicity and long-term transgenic expression. Clinical studies on gene therapy based on the AAV vector have been extensively developed in recent years and are being touted as the next-generation treatm...