RNA Interference-Based Therapy for Spinocerebellar Ataxia Type 7 Retinal Degeneration

Spinocerebellar ataxia type 7 (SCA7) is an autosomal dominant neurodegenerative disease characterized by loss of motor coordination and retinal degeneration with no current therapies in the clinic. The causative mutation is an expanded CAG repeat in the ataxin-7 gene whose mutant protein product cau...

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Bibliographic Details
Main Authors: Ramachandran, Pavitra S., Bhattarai, Sajag, Singh, Pratibha, Boudreau, Ryan L., Thompson, Stewart, LaSpada, Albert R., Drack, Arlene V., Davidson, Beverly L.
Format: Online
Language:English
Published: Public Library of Science 2014
Online Access:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3997397/