Long-term RNA interference gene therapy in a dominant retinitis pigmentosa mouse model

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...

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Hlavní autoři: Jiang, Li, Zhang, Houbin, Dizhoor, Alexander M., Boye, Shannon E., Hauswirth, William W., Frederick, Jeanne M., Baehr, Wolfgang
Médium: Artigo
Jazyk:Inglês
Vydáno: National Academy of Sciences 2011
Témata:
Biological Sciences
On-line přístup:https://ncbi.nlm.nih.gov/pmc/articles/PMC3215008/
https://ncbi.nlm.nih.gov/pubmed/22042849
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1073/pnas.1112758108
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