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Suppression of rds Expression by siRNA and Gene Replacement Strategies for Gene Therapy Using rAAV Vector

Small interfering RNA (siRNA) is a promising tool for the treatment of dominant diseases. Autosomal dominant eye disease like retinitis pigmentosa, are a leading cause of blindness. Mutations in rds/peripherin lead to the degeneration of photoreceptors and are associated with several autosomal retin...

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Bibliografiske detaljer
Main Authors: Petrs-Silva, Hilda, Yasumura, Douglas, Matthes, Michael T., LaVail, Matthew M., Lewin, Alfred S., Hauswirth, William W.
Format: Artigo
Sprog:Inglês
Udgivet: 2012
Fag:
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC3556273/
https://ncbi.nlm.nih.gov/pubmed/22183336
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1007/978-1-4614-0631-0_29
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