ASOs are an effective treatment for disease-associated oligodendrocyte signatures in premanifest and symptomatic SCA3 mice

We report a severe, but modifiable, deficit in oligodendrocyte maturation caused by the toxic gain-of-function of mutant ATXN3 early in SCA3 disease that is transcriptionally, biochemically, and functionally rescued with anti-ATXN3 ASO. Our results highlight the promising use of an ASO therapy across neurodegenerative diseases that requires glial targeting in addition to affected neuronal populations.PMID:38429929 | DOI:10.1016/j.ymthe.2024.02.033

Source: Molecular Medicine - March 2, 2024 Category: Molecular Biology Authors: Kristen H Schuster Annie J Zalon Danielle M DiFranco Alexandra F Putka Nicholas R Stec Sabrina I Jarrah Arsal Naeem Zaid Haque Hanrui Zhang Yuanfang Guan Hayley S McLoughlin Source Type: research