Antisense Oligonucleotide-Mediated Removal of the Polyglutamine Repeat in Spinocerebellar Ataxia Type 3 Mice

• Principais autores: Lodewijk J.A. Toonen (Autor), Frank Rigo (Autor), Haico van Attikum (Autor), Willeke M.C. van Roon-Mom (Autor)
• Formato: Livro
• Publicado em: Elsevier, 2017-09-01T00:00:00Z.
• Assuntos: article
• Acesso em linha: Connect to this object online.

Spinocerebellar ataxia type 3 (SCA3) is a currently incurable neurodegenerative disorder caused by a CAG triplet expansion in exon 10 of the ATXN3 gene. The resultant expanded polyglutamine stretch in the mutant ataxin-3 protein causes a gain of toxic function, which eventually leads to neurodegener...