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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Publicat a:Sci Transl Med
Autors principals: 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.
Format: Artigo
Idioma:Inglês
Publicat: 2018
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Article
Accés en línia: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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