Antisense oligonucleotides targeting mutant Ataxin-7 restore visual function in a mouse model of spinocerebellar ataxia type 7

Spinocerebellar ataxia type 7 (SCA7) is an autosomal dominant neurodegenerative disorder characterized by cerebellar and retinal degeneration, and is caused by a CAG-polyglutamine repeat expansion in the ATAXIN-7 gene. SCA7 patients develop progressive cone-rod dystrophy, typically resulting in blin...

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Publicado no:Sci Transl Med
Main Authors: Niu, Chenchen, Prakash, Thazah P., Kim, Aneeza, Quach, John L., Huryn, Laryssa A., Yang, Yuechen, Lopez, Edith, Jazayeri, Ali, Hung, Gene, Sopher, Bryce L., Brooks, Brian P., Swayze, Eric E., Bennett, C. Frank, La Spada, Albert R.
Formato: Artigo
Idioma:Inglês
Publicado em: 2018
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Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC6411060/
https://ncbi.nlm.nih.gov/pubmed/30381411
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1126/scitranslmed.aap8677
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