Marin M, Noël D, Piechaczyk M
Institut de Génétique Moléculaire, CNRS-UMR, Montpellier, France.
Mol Med Today. 1997 Sep;3(9):396-403. doi: 10.1016/S1357-4310(97)01095-2.
Efficient human gene therapy will rely on efficient gene delivery systems. To date, recombinant, engineered retroviruses are the most widely used vectors for stable clinical gene transfer. However, this technology still suffers from a number of drawbacks that limit its application. The ability to target cells of therapeutic interest, in particular, would improve both the efficiency and the safety of retroviral vectors. It might also allow the development of new animal models of human diseases and probably extend the scope of gene therapy itself.
高效的人类基因治疗将依赖于高效的基因递送系统。迄今为止,重组工程逆转录病毒是临床基因稳定转移中使用最广泛的载体。然而,这项技术仍然存在一些限制其应用的缺点。特别是,靶向治疗相关细胞的能力将提高逆转录病毒载体的效率和安全性。它还可能有助于开发人类疾病的新动物模型,并可能扩展基因治疗本身的范围。