Oliveira Ana V, Rosa da Costa Ana M, Silva Gabriela A
Center for Biomedical Research (CBMR), University of Algarve, Faro 8005-139, Portugal.
Department of Chemistry and Pharmacy, University of Algarve, Faro 8005-139, Portugal; Algarve Chemistry Research Centre (CIQA), University of Algarve, Faro 8005-139, Portugal.
Mater Sci Eng C Mater Biol Appl. 2017 Aug 1;77:1275-1289. doi: 10.1016/j.msec.2017.04.068. Epub 2017 Apr 18.
The success of gene therapy relies on efficient gene transfer and stable transgene expression. The in vivo efficiency is determined by the delivery vector, route of administration, therapeutic gene, and target cells. While some requirements are common to several strategies, others depend on the target disease and transgene product. Consequently, it is unlikely that a single system is suitable for all applications. This review examines current gene therapy strategies, focusing on non-viral approaches and the use of natural polymers with the eye, and particularly the retina, as their gene delivery target.
基因治疗的成功依赖于高效的基因转移和稳定的转基因表达。体内效率由递送载体、给药途径、治疗性基因和靶细胞决定。虽然一些要求是几种策略共有的，但其他要求则取决于靶疾病和转基因产物。因此，不太可能有单一系统适用于所有应用。本综述考察了当前的基因治疗策略，重点关注非病毒方法以及以眼睛，尤其是视网膜作为基因递送靶点的天然聚合物的应用。
