腺相关病毒载体整合的染色体效应。

Chromosomal effects of adeno-associated virus vector integration.

作者信息

Miller Daniel G, Rutledge Elizabeth A, Russell David W

机构信息

Department of Medicine, University of Washington, Seattle, Washington, 98195, USA.

出版信息

Nat Genet. 2002 Feb;30(2):147-8. doi: 10.1038/ng824. Epub 2002 Jan 22.

DOI:10.1038/ng824

PMID:11799395

Abstract

Adeno-associated virus (AAV) vectors are currently being used in several clinical gene-therapy trials (see the NIH OBA Human Gene Transfer Clinical Trials Database); however, little is known about the chromosomal effects of vector integration. Here we report that integrated vector proviruses are associated with chromosomal deletions and other rearrangements and are frequently located on chromosome 19 (although not at the wildtype AAV integration site).

摘要

腺相关病毒（AAV）载体目前正用于多项临床基因治疗试验（见美国国立卫生研究院生物技术评估办公室人类基因转移临床试验数据库）；然而，关于载体整合的染色体效应却知之甚少。在此我们报告，整合的载体前病毒与染色体缺失及其他重排有关，并且经常位于19号染色体上（尽管并非位于野生型AAV整合位点）。