Ontology highlight
ABSTRACT:
SUBMITTER: Anderson BR
PROVIDER: S-EPMC7033438 | biostudies-literature | 2020 Mar
REPOSITORIES: biostudies-literature
Molecular therapy. Nucleic acids 20200121
Hundreds of dominant-negative myosin mutations have been identified that lead to hypertrophic cardiomyopathy, and the biomechanical link between mutation and disease is heterogeneous across this patient population. To increase the therapeutic feasibility of treating this diverse genetic population, we investigated the ability of locked nucleic acid (LNA)-modified antisense oligonucleotides (ASOs) to selectively knock down mutant myosin transcripts by targeting single-nucleotide polymorphisms (SN ...[more]