Article
Silencing gene therapy for mutant membrane, secretory, and lipid proteins in retinitis pigmentosa (RP).
Medical hypotheses - 1 Jan 2008
Shinohara Toshimichi, Mulhern Michael L, Madson Christian J
Abstract excerpt
A mutation in retinal photoreceptor-specific proteins causes nearly 50% of retinitis pigmentosa (RP) cases; the other 50% is called sporadic RP, the etiology of which is unknown. To alleviate RP development, gene therapies, including insertion of a wild type gene or replacement of mutant genes, have been conducted in animal models. The result was not always satisfactory. We found that a mutant misfolded rhodopsin...
Read the complete abstract on PubMedTopics
Share this publication in a Topic to start or enrich a Post.
