Ontology highlight
ABSTRACT:
SUBMITTER: Tanner MK
PROVIDER: S-EPMC7913682 | biostudies-literature | 2021 Feb
REPOSITORIES: biostudies-literature
Tanner Matthew K MK Tang Zhenzhi Z Thornton Charles A CA
Nucleic acids research 20210201 4
Biomarker-driven trials hold promise for therapeutic development in chronic diseases, such as muscular dystrophy. Myotonic dystrophy type 1 (DM1) involves RNA toxicity, where transcripts containing expanded CUG-repeats (CUGexp) accumulate in nuclear foci and sequester splicing factors in the Muscleblind-like (Mbnl) family. Oligonucleotide therapies to mitigate RNA toxicity have emerged but reliable measures of target engagement are needed. Here we examined muscle transcriptomes in mouse models o ...[more]