Deciphering the Complex Molecular Pathogenesis of Myotonic Dystrophy Type 1 through Omics Studies.
Int J Mol Sci
; 23(3)2022 Jan 27.
Article
in En
| MEDLINE
| ID: mdl-35163365
ABSTRACT
Omics studies are crucial to improve our understanding of myotonic dystrophy type 1 (DM1), the most common muscular dystrophy in adults. Employing tissue samples and cell lines derived from patients and animal models, omics approaches have revealed the myriad alterations in gene and microRNA expression, alternative splicing, 3' polyadenylation, CpG methylation, and proteins levels, among others, that contribute to this complex multisystem disease. In addition, omics characterization of drug candidate treatment experiments provides crucial insight into the degree of therapeutic rescue and off-target effects that can be achieved. Finally, several innovative technologies such as single-cell sequencing and artificial intelligence will have a significant impact on future DM1 research.
Key words
Full text:
1
Database:
MEDLINE
Main subject:
Computational Biology
/
Myotonic Dystrophy
Type of study:
Etiology_studies
Limits:
Adult
/
Animals
/
Humans
Language:
En
Year:
2022
Type:
Article