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

• Principais autores: Stefan Hauser (Autor), Jacob Helm (Autor), Melanie Kraft (Autor), Milena Korneck (Autor), Jeannette Hübener-Schmid (Autor), Ludger Schöls (Autor)
• Formato: Livro
• Publicado em: Elsevier, 2022-03-01T00:00:00Z.
• Assuntos: article
• Acesso em linha: Connect to this object online.

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...