Gene therapy in animal models of autosomal dominant retinitis pigmentosa.
Year: 2012
Type: article
Abstract: Gene therapy for dominantly inherited genetic disease is more difficult than gene-based therapy for recessive disorders, which can be treated with gene supplementation. Treatment of dominant disease may require gene supplementation partnered with suppression of the expression of the mutant gene either at the DNA level, by gene repair, or at the RNA level by RNA interference or transcriptional repression. In this review, we examine some of the gene delivery approaches used to treat animal models of autosomal dominant retinitis pigmentosa, focusing on those models associated with mutations in the gene for rhodopsin. We conclude that combinatorial approaches have the greatest promise for success.
Source: PubMed Central
Authors: Brian Rossmiller, Haoyu Mao, Alfred S Lewin
Institution: University of Florida
Language: English
Subfield: Ophthalmology
Field: Medicine
Domain: Health Sciences
Open Access status: green
Funder: National Eye Institute
OpenAlex ID: W1492014360