current concepts in the treatment of retinitis pigmentosa目前治疗视网膜色素变性的概念.pdfVIP

Hindawi Publishing Corporation Journal of Ophthalmology Volume 2011, Article ID 753547, 8 pages doi:10.1155/2011/753547 Review Article Current Concepts in the Treatment of Retinitis Pigmentosa Maria A. Musarella1 and Ian M. MacDonald2 1 Department of Ophthalmology, SUNY Downstate Medical Center, Brooklyn, NY 11203, USA 2 Department of Ophthalmology, Royal Alexandra Hospital, University of Alberta, 10240 Kingsway Avenue, Rm. 2319, Edmonton, Canada AB T5H 3V9 Correspondence should be addressed to Ian M. MacDonald, macdonal@ualberta.ca Received 24 May 2010; Accepted 16 September 2010 Academic Editor: Muna Naash Copyright © 2011 M. A. Musarella and I. M. MacDonald. This is an open access article distributed under the Creative Commons Attribution License, which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited. Inherited retinal degenerations, including retinitis pigmentosa (RP) and Leber congenital amaurosis (LCA), affect 1 in 4000 individuals in the general population. A majority of the genes which are mutated in these conditions are expressed in either photoreceptors or the retinal pigment epithelium (RPE). There is considerable variation in the clinical severity of these conditions; the most severe being autosomal recessive LCA, a heterogeneous retinal degenerative disease and the commonest cause of congenital blindness in children. Here, we discuss all the potential treatments that are now available for retinal degeneration. A number of therapeutic avenues are being explored based on our knowledge of the pathophysiology of retinal degeneration derived from research on animal models, including: gene therapy, antiapoptosis agents, neurotrophic factors, and dietary s