Gene therapy in animal models of autosomal dominant retinitis pigmentosa.
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.
Department of Molecular Genetics and Microbiology, University of Florida, Gainesville, FL 32611, USA.
Brian
B
Rossmiller
Haoyu
H
Mao
Alfred S
AS
Lewin
D000256:Adenoviridae / Q000235:genetics; D000818:Animals; D004195:Disease Models, Animal; D015870:Gene Expression; D018014:Gene Transfer Techniques; D005799:Genes, Dominant; D015316:Genetic Therapy; D005822:Genetic Vectors; D006801:Humans; D051379:Mice; D009154:Mutation; D034622:RNA Interference; D012174:Retinitis Pigmentosa / Q000235:genetics* / Q000473:pathology / Q000628:therapy*; D012243:Rhodopsin / Q000235:genetics*; D014158:Transcription, Genetic
D012243:Rhodopsin