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Development of gene therapy-based strategies for the treatment of eye diseases

✍ Scribed by Piroska E. Rakoczy; Wei-Yong Shen; May Lai; Fabienne Rolling; Ian J. Constable

Publisher: John Wiley and Sons
Year: 1999
Tongue: English
Weight: 289 KB
Volume: 46
Category: Article
ISSN: 0272-4391
DOI: 10.1002/(sici)1098-2299(199903/04)46:3/4<277::aid-ddr13>3.0.co;2-b

No coin nor oath required. For personal study only.

✦ Synopsis

Gene therapy, which is the deliberate transfer of DNA for therapeutic purposes, can be defined as the in situ production of a therapeutical material within the diseased cells. The eye is an ideal target for gene therapy, as it is confined to a small space, separated by the retina-blood barrier from other organs, there are a limited number of cells present, and the endpoint can be optically monitored. Following subretinal injection, recombinant adenovirus and recombinant adeno-associated viruses successfully transduced retinal cells. Of the retinal cells, retinal pigment epithelial cells were transduced with the highest efficiency. The expression of the transgene was transient in adenovirus-injected eyes. In contrast, the expression of the transgene in adeno-associated virus-injected eyes was present throughout the 3-month examination period. These observations suggest that gene therapy with adeno-associated virus could be a suitable approach for the treatment of genetic diseases of the retinal pigment epithelium and adenoviruses for the transient regulation of gene expression in retinal pigment epithelial cells.

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