Antisense oligonucleotide therapy rescues aggresome formation in a novel Spinocerebellar Ataxia type 3 human embryonic stem cell line.

Spinocerebellar ataxia type 3 (SCA3) is a fatal, late-onset neurodegenerative disorder characterized by selective neuropathology in the brainstem, cerebellum, spinal cord, and substantia nigra. Here we report the first NIH-approved human embryonic stem cell (hESC) line derived from an embryo harbori...

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Argitaratua izan da:Stem Cell Res
Egile Nagusiak: Moore, Lauren R., Keller, Laura, Bushart, David D., Delatorre, Rodrigo, Li, Dorcas, McLoughlin, Hayley S., Costa, Maria do Carmo, Shakkottai, Vikram G., Smith, Gary D., Paulson, Henry L.
Formatua: Artigo
Hizkuntza:Inglês
Argitaratua: 2019
Gaiak:
Article
Sarrera elektronikoa:https://ncbi.nlm.nih.gov/pmc/articles/PMC6736695/
https://ncbi.nlm.nih.gov/pubmed/31374463
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.scr.2019.101504
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