Non-viral strategies for ocular gene delivery

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.

Abstract

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.

Keywords: Eye; Gene therapy; Non-viral vectors; Retina.

Publication types

  • Review

MeSH terms

  • Gene Transfer Techniques*
  • Genetic Therapy
  • Genetic Vectors
  • Humans
  • Transgenes