Shelling A N, Smith M G
Biochemistry Department, University of Otago, Dunedin, New Zealand.
Gene Ther. 1994 May;1(3):165-9.
Human adeno-associated virus (AAV) integrates specifically into a site (AAVS1) on chromosome 19q13.3-qter. We report that both transfected and infected AAV-vectors will target a foreign gene preferentially to AAVS1. Of 11 infected cell lines studied, nine (82%) showed integration of the neomycin resistance marker into AAVS1, which confirms the findings of previous investigations. We also show site-specific integration (nine out of 12 cell lines, 75%) following transfection of an AAV vector containing the neomycin gene. The ability of transfected AAV sequences to preferentially target and express selectable genes at a known site in the human genome extends the potential use of AAV as a vector in gene therapy.
人类腺相关病毒（AAV）特异性整合到19号染色体q13.3 - qter区域的一个位点（AAVS1）。我们报告，无论是转染的还是感染的AAV载体都会优先将外源基因靶向AAVS1。在所研究的11个感染细胞系中，9个（82%）显示新霉素抗性标记整合到AAVS1中，这证实了先前研究的结果。我们还展示了转染含有新霉素基因的AAV载体后出现的位点特异性整合（12个细胞系中的9个，75%）。转染的AAV序列优先靶向人类基因组中一个已知位点并表达可选择基因的能力扩展了AAV作为基因治疗载体的潜在用途。
