Photoreceptor cell rescue in retinal degeneration (rd) mice by in vivo gene therapy
Copyright policy )Photoreceptor cell rescue in retinal degeneration (rd) mice by in vivo gene therapy
Mutations in the beta subunit of the cGMP phosphodiesterase gene (beta PDE) can cause a recessively inherited retinal degeneration in several species, including mice, dogs and humans. We tested the possibility of altering the course of retinal degeneration in the rd mouse through subretinal injection of a recombinant replication-defective adenovirus that contains the murine cDNA for wild-type (beta PDE, Ad.CMV beta PDE. Subretinal injection of Ad.CMV beta PDE results in beta PDE transcripts and increased PDE activity and delays photoreceptor cell death by six weeks. The findings demonstrate cell rescue by in vivo gene transfer, thus supporting the feasibility of treating an inherited retinal degeneration by somatic gene therapy.
- King’s University United States
- Pfizer (United States) United States
- University of Pennsylvania United States
- Human Gene Therapy Research Institute United States
- University of Chicago United States
Phosphoric Diester Hydrolases, Genetic Vectors, Homozygote, Retinal Degeneration, Gene Transfer Techniques, Cytomegalovirus, Mice, Inbred Strains, Eye, Cyclic Nucleotide Phosphodiesterases, Type 3, Retina, Adenoviridae, Injections, Disease Models, Animal, Mice, Gene Expression Regulation, 3',5'-Cyclic-AMP Phosphodiesterases, Animals, Tissue Distribution
Phosphoric Diester Hydrolases, Genetic Vectors, Homozygote, Retinal Degeneration, Gene Transfer Techniques, Cytomegalovirus, Mice, Inbred Strains, Eye, Cyclic Nucleotide Phosphodiesterases, Type 3, Retina, Adenoviridae, Injections, Disease Models, Animal, Mice, Gene Expression Regulation, 3',5'-Cyclic-AMP Phosphodiesterases, Animals, Tissue Distribution
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