Inherited retinal degenerations: therapeutic prospects.

Abstract:

:Retinitis pigmentosa (RP) is a heterogeneous group of inherited retinal degenerative diseases, characterized by the progressive death of rod and cone photoreceptors. A tremendous genetic heterogeneity is associated with the RP phenotype. Most mutations affect rods selectively and, through an unknown pathway, cause the rod cells to die by apoptosis. Cones, on the other hand, are seldom directly affected by the identified mutations, and yet, in many cases, they degenerate secondarily to rods, which accounts for loss of central vision and complete blindness. Many animal models of RP are available and have led to a better understanding of the disease and to the development of therapeutic strategies aimed at curing the specific genetic disorder (gene therapy), slowing down or even stopping the process of photoreceptor degeneration (growth factors or calcium blockers applications, vitamin supplementation), preserving the cones implicated in the central visual function (identification of endogenous cone viability factors) or even replacing the lost cells (transplantation, use of stem or precursor cells). Still, many obstacles will need to be overcome before most of these strategies can be applied to humans. In this review, we describe the different therapeutic strategies being studied worldwide and report the latest results in this field.

journal_name

Biol Cell

journal_title

Biology of the cell

authors

Delyfer MN,Léveillard T,Mohand-Saïd S,Hicks D,Picaud S,Sahel JA

doi

10.1016/j.biolcel.2004.01.006

subject

Has Abstract

pub_date

2004-05-01 00:00:00

pages

261-9

issue

4

eissn

0248-4900

issn

1768-322X

pii

S0248490004000346

journal_volume

96

pub_type

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