Suppression and replacement gene therapy for autosomal dominant disease in a murine model of dominant retinitis pigmentosa.

ABSTRACT: For dominantly inherited disorders development of gene therapies, targeting the primary genetic lesion has been impeded by mutational heterogeneity. An example is rhodopsin-linked autosomal dominant retinitis pigmentosa with over 150 mutations in the rhodopsin gene. Validation of a mutation-independent suppression and replacement gene therapy for this disorder has been undertaken. The therapy provides a means of correcting the genetic defect in a mutation-independent manner thereby circumventing the mutational diversity. Separate adeno-associated virus (AAV) vectors were used to deliver an RNA interference (RNAi)-based rhodopsin suppressor and a codon-modified rhodopsin replacement gene resistant to suppression due to nucleotide alterations at degenerate positions over the RNAi target site

SUBMITTER: Millington-Ward S 

PROVIDER: S-EPMC3070095 | biostudies-literature | 2011 Apr

REPOSITORIES: biostudies-literature