MATERIALS AND METHODS FOR TREATMENT OF RETINAL DISEASES

摘要

The subject invention provides materials and methods for efficient, specific expression of proteins in retinal photoreceptor cells. Specifically, the constructs are composed of an adeno-associated vital vector containing a rod or cone-opsin promoter. These materials and methods can be used in therapies for retinal diseases. In one embodiment, ribozymes which degrade mutant mRNA are used to treat retinitis pigmentosa.