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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.

Non-viral strategies for ocular gene delivery.

Author information

1
Center for Biomedical Research (CBMR), University of Algarve, Faro 8005-139, Portugal.
2
Department of Chemistry and Pharmacy, University of Algarve, Faro 8005-139, Portugal; Algarve Chemistry Research Centre (CIQA), University of Algarve, Faro 8005-139, Portugal.
3
CEDOC, NOVA Medical School, Faculdade de Ciências Médicas, Universidade Nova de Lisboa, Campo Mártires da Pátria 130, 1169-056 Lisboa, Portugal. Electronic address: gabriela.silva@nms.unl.pt.

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

PMID:
28532005
DOI:
10.1016/j.msec.2017.04.068
[Indexed for MEDLINE]

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