Cosset F L, Russell S J
Centre de Génétique Moléculaire et Cellulaire, CNRS UMR5534, Université Claude-Bernard Lyon-1, Villeurbanne, France.
Gene Ther. 1996 Nov;3(11):946-56.
Most of the clinical gene therapy trials that have been initiated to date have employed ex vivo strategies in which cells are genetically modified outside the body and reimplanted. The ability to deliver genes accurately and efficiently to selected target cell populations in vivo would greatly expand the scope of gene therapy, but current vectors are not well suited to this task. Here we review recent attempts to develop retroviral vectors incorporating engineered envelope glycoproteins that are capable of delivering their genes in a highly specific manner to selected human target cells.
迄今为止启动的大多数临床基因治疗试验都采用了体外策略，即先在体外对细胞进行基因改造，然后再重新植入体内。能够在体内将基因准确、高效地传递到选定的靶细胞群体，将极大地扩展基因治疗的范围，但目前的载体并不适合这项任务。在此，我们综述了近期开发逆转录病毒载体的尝试，这些载体包含经过工程改造的包膜糖蛋白，能够以高度特异性的方式将其基因传递到选定的人类靶细胞。
