Ontology highlight
ABSTRACT:
SUBMITTER: Lim KRQ
PROVIDER: S-EPMC7368245 | biostudies-literature | 2020 Jul
REPOSITORIES: biostudies-literature
Lim Kenji Rowel Q KRQ Maruyama Rika R Echigoya Yusuke Y Nguyen Quynh Q Zhang Aiping A Khawaja Hunain H Sen Chandra Sreetama S Jones Takako T Jones Peter P Chen Yi-Wen YW Yokota Toshifumi T
Proceedings of the National Academy of Sciences of the United States of America 20200629 28
Facioscapulohumeral muscular dystrophy (FSHD), characterized by progressive muscle weakness and deterioration, is genetically linked to aberrant expression of <i>DUX4</i> in muscle. DUX4, in its full-length form, is cytotoxic in nongermline tissues. Here, we designed locked nucleic acid (LNA) gapmer antisense oligonucleotides (AOs) to knock down <i>DUX4</i> in immortalized FSHD myoblasts and the <i>FLExDUX4</i> FSHD mouse model. Using a screening method capable of reliably evaluating the knockdo ...[more]