Adenine base editing of the <i>DUX4</i> polyadenylation signal for targeted genetic therapy in facioscapulohumeral muscular dystrophy.

Adenine base editing of the DUX4 polyadenylation signal for targeted genetic therapy in facioscapulohumeral muscular dystrophy.

Sikrová, Darina; Cadar, Vlad A; Ariyurek, Yavuz; Laros, Jeroen F J; Balog, Judit; van der Maarel, Silvère M.

Afiliação

Sikrová D; Department of Human Genetics, Leiden University Medical Center, 2333 ZC Leiden, the Netherlands.
Cadar VA; Leiden University, 2300 RA Leiden, the Netherlands.
Ariyurek Y; Department of Human Genetics, Leiden University Medical Center, 2333 ZC Leiden, the Netherlands.
Laros JFJ; Leiden Genome Technology Center, Leiden University Medical Center, 2333 ZC Leiden, the Netherlands.
Balog J; Department of Human Genetics, Leiden University Medical Center, 2333 ZC Leiden, the Netherlands.
van der Maarel SM; Department of Clinical Genetics, Leiden University Medical Center, 2333 ZC Leiden, the Netherlands.

Mol Ther Nucleic Acids ; 25: 342-354, 2021 Sep 03.

Article em En | MEDLINE | ID: mdl-34484861