Antisense oligonucleotides as a potential treatment for brain deficits observed in myotonic dystrophy type 1.

ABSTRACT: Myotonic dystrophy, or dystrophia myotonica type 1 (DM1), is a multi-systemic disorder and is the most common adult form of muscular dystrophy. It affects not only muscles but also many organs, including the brain. Cerebral impairments include cognitive deficits, daytime sleepiness, and loss of visuospatial and memory functions. The expression of mutated transcripts with CUG repeats results in a gain of toxic mRNA function. The antisense oligonucleotide (ASO) strategy to treat DM1 brain deficits is limited by the fact that ASOs do not cross the blood-brain barrier after systemic administration, indicating that other methods of delivery should be considered. ASO technology has emerged as a powerful tool for developing potential new therapies for a wide variety of human diseases, and its pot

SUBMITTER: Ait Benichou S 

PROVIDER: S-EPMC9750879 | biostudies-literature | 2022 Dec

REPOSITORIES: biostudies-literature