[Survival factors in the treatment of hereditary retinal degeneration]. / Uberlebensfaktoren in der Therapie erblicher Netzhautdegenerationen.

ABSTRACT

Hereditary retinal degeneration is characterized by apoptotic photoreceptor loss, a process governed by intricate molecular interplay and initiated when proapoptotic signals predominate in the individual cell. Identification of molecules involved and their actions has paved the way for testing the ones with anti-apoptotic functions in models of inherited retinal degeneration. Many of these factors are able to slow the course of the degeneration. However, to date no such treatment has been able to stop or even prevent the devolution of the disorder. Moreover, preservation of morphology does not necessarily correlate with preservation of ERG function. Deepened understanding of the pro- and anti-apoptotic networks is clearly needed for survival factors to be feasible for therapy in humans. In comparison, in a dog model of Leber's congenital amaurosis gene therapy could establish retinal function, thus supplying proof of efficacy of the method.