Ontology highlight
ABSTRACT:
SUBMITTER: Tran H
PROVIDER: S-EPMC8861976 | biostudies-literature | 2022 Jan
REPOSITORIES: biostudies-literature
Tran Hélène H Moazami Michael P MP Yang Huiya H McKenna-Yasek Diane D Douthwright Catherine L CL Pinto Courtney C Metterville Jake J Shin Minwook M Sanil Nitasha N Dooley Craig C Puri Ajit A Weiss Alexandra A Wightman Nicholas N Gray-Edwards Heather H Marosfoi Miklos M King Robert M RM Kenderdine Thomas T Fabris Daniele D Bowser Robert R Watts Jonathan K JK Brown Robert H RH
Nature medicine 20211223 1
Expansions of a G<sub>4</sub>C<sub>2</sub> repeat in the C9ORF72 gene are the most common genetic cause of amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD), two devastating adult-onset neurodegenerative disorders. Using C9-ALS/FTD patient-derived cells and C9ORF72 BAC transgenic mice, we generated and optimized antisense oligonucleotides (ASOs) that selectively blunt expression of G<sub>4</sub>C<sub>2</sub> repeat-containing transcripts and effectively suppress tissue levels ...[more]