Allele-specific targeting of mutant ataxin-3 by antisense oligonucleotides in SCA3-iPSC-derived neurons

Spinocerebellar ataxia type 3 (SCA3) is caused by an expanded polyglutamine stretch in ataxin-3. While wild-type ataxin-3 has important functions, e.g., as a deubiquitinase, downregulation of mutant ataxin-3 is likely to slow down the course of this fatal disease. We established a screening platform...

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Detalles Bibliográficos
Autores principales: Hauser, Stefan, Helm, Jacob, Kraft, Melanie, Korneck, Milena, Hübener-Schmid, Jeannette, Schöls, Ludger
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2021
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8649108/
https://www.ncbi.nlm.nih.gov/pubmed/34938609
http://dx.doi.org/10.1016/j.omtn.2021.11.015

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8649108/
https://www.ncbi.nlm.nih.gov/pubmed/34938609
http://dx.doi.org/10.1016/j.omtn.2021.11.015

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