Antisense oligonucleotide therapy targeted against ATXN3 improves potassium channel-mediated Purkinje neuron dysfunction in spinocerebellar ataxia type 3

Spinocerebellar ataxia type 3 (SCA3) is the second-most common CAG repeat disease, caused by a glutamine-encoding expansion in the ATXN3 protein. SCA3 is characterized by spinocerebellar degeneration leading to progressive motor incoordination and early death. Previous studies suggest that potassium...

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Publicado en:Cerebellum
Main Authors: Bushart, David D., Zalon, Annie J., Zhang, Hongjiu, Morrison, Logan M., Guan, Yuanfang, Paulson, Henry L., Shakkottai, Vikram G., McLoughlin, Hayley S.
Formato: Artigo
Idioma:Inglês
Publicado: 2021
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Article
Acceso en liña:https://ncbi.nlm.nih.gov/pmc/articles/PMC7930886/
https://ncbi.nlm.nih.gov/pubmed/32789747
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1007/s12311-020-01179-7
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