Skip to Main Content

Paper Details

Adenine base editing of the <i>DUX4</i> polyadenylation signal for targeted genetic therapy in facioscapulohumeral muscular dystrophy.
Mol Ther Nucleic Acids
12
2021
Adenine, CRISPR, Cas9, D4Z4, D4Z4 repeat, D4Z4-encoded DUX4 gene, DNA, DUX4, DUX4 gene, DUX4 mRNA, DUX4 polyadenylation signal, FSHD, Facioscapulohumeral muscular dystrophy, adenine, chromatin, cis-, exon three, facioscapulohumeral muscular dystrophy, full-length DUX4 mRNA, genetic diseases, immortalized myoblasts, nucleotide, polyadenylation signal, postmitotic cells, regulatory element, skeletal, skeletal muscle, somatic cells, transcriptional target genes
Author NameAffiliation
Yavuz AriyurekLeiden University Medical Center
Yavuz AriyurekLeiden University Medical Center
Jeroen F J LarosLeiden University Medical Center
Jeroen F J LarosLeiden University Medical Center
Jeroen F J LarosNational Institute for Public Health and the Environment (RIVM)
  • 1 - 5

Datasets

Release 1.2