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Journal article
Long-term RNA interference gene therapy in a dominant retinitis pigmentosa mouse model
Proceedings of the National Academy of Sciences - PNAS, v 108(45), pp 18476-18481
08 Nov 2011
PMID: 22042849
Featured in Collection : UN Sustainable Development Goals @ Drexel
Abstract
RNA interference (RNAi) gene silencing is a potential therapeutic strategy for dominant retinal degeneration disorders. We used self-complementary (sc) AAV2/8 vector to develop an RNAi-based gene therapy in a dominant retinal degeneration mouse model expressing bovine GCAP1(Y99C). We established an in vitro shRNA screening assay based on EGFP-tagged bovine GCAP1, and identified a shRNA that effectively silenced the bovine GCAP1 transgene with ∼80% efficiency. Subretinal injection of scAAV2/8 carrying shRNA expression cassette showed robust expression as early as 1 wk after injection. The gene silencing significantly improved photoreceptor survival, delayed disease onset, and increased visual function. Our results provide a promising strategy toward effective RNAi-based gene therapy by scAAV2/8 delivery for dominant retinal diseases.
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Details
- Title
- Long-term RNA interference gene therapy in a dominant retinitis pigmentosa mouse model
- Creators
- Li Jiang - University of UtahHoubin Zhang - University of UtahAlexander M. Dizhoor - Salus UniversityShannon E. Boye - University of Florida College of MedicineWilliam W. Hauswirth - University of FloridaJeanne M. Frederick - University of UtahWolfgang Baehr - University of Utah
- Publication Details
- Proceedings of the National Academy of Sciences - PNAS, v 108(45), pp 18476-18481
- Publisher
- National Academy of Sciences
- Number of pages
- 6
- Resource Type
- Journal article
- Language
- English
- Academic Unit
- Neurobiology and Anatomy
- Web of Science ID
- WOS:000296700000064
- Scopus ID
- 2-s2.0-81055141290
- Other Identifier
- 991022035114104721
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- Collaboration types
- Domestic collaboration
- Web of Science research areas
- Biochemistry & Molecular Biology