Department of Molecular and Cellular Biology, Beckman Research Institute of City of Hope, Duarte, CA 91010, USA.
Hum Mol Genet. 2011 Apr 15;20(R1):R100-7. doi: 10.1093/hmg/ddr160. Epub 2011 Apr 19.
Until recently, progress in ex vivo gene therapy (GT) for human immunodeficiency virus-1 (HIV-1) treatment has been incremental. Long-term HIV-1 remission in a patient who received a heterologous stem cell transplant for acquired immunodeficiency syndrome-related lymphoma from a CCR5(-/-) donor, even after discontinuation of conventional therapy, has energized the field. We review the status of current approaches as well as future directions in the areas of therapeutic targets, combinatorial strategies, vector design, introduction of therapeutics into stem cells and enrichment/expansion of gene-modified cells. Finally, we discuss recent advances towards clinical application of HIV-1 GT.
直到最近，用于人类免疫缺陷病毒 1（HIV-1）治疗的体外基因治疗（GT）的进展一直是渐进的。一位患有获得性免疫缺陷综合征相关淋巴瘤的患者接受了来自 CCR5（-/-）供体的异基因干细胞移植，即使在停止常规治疗后，HIV-1 仍长期缓解，这激发了该领域的研究热情。我们回顾了目前在治疗靶点、组合策略、载体设计、将治疗药物引入干细胞以及基因修饰细胞的富集/扩增等领域的现状和未来方向。最后，我们讨论了 HIV-1 GT 临床应用的最新进展。
