Applying genome-wide CRISPR-Cas9 screens for therapeutic discovery in facioscapulohumeral muscular dystrophy
Angela Lek(Muscular Dystrophy Association), Louis M. Kunkel(Boston Children's Hospital), Monkol Lek(Massachusetts General Hospital), Peter L. Jones(University of Nevada, Reno), Alec M. DeSimone(University of Massachusetts Chan Medical School), Anna Pakuła(Boston Children's Hospital), Keryn G. Woodman(Yale University), Tracy Zhang(Boston Children's Hospital), Andrew Kodani(St. Jude Children's Research Hospital), Lillian Mead(Boston Children's Hospital), Oliver D. King(Boston Biomedical Research Institute), Shushu Huang(Yale University), Justin Cohen(Yale University), James R. Conner(Boston Children's Hospital), Vincent Ho(Yale University), Kathryn R. Wagner(Kennedy Krieger Institute), Neville E. Sanjana(New York Genome Center)
Cited by 79
Related Papers
Exploring the phenotypic consequences of tissue specific gene expression variation inferred from GWAS summary statistics
|Nature Communications|2018|1.2k
The molecular basis for Duchenne versus Becker muscular dystrophy: correlation of severity with type of deletion.
|PubMed|1989|961
Improving genetic diagnosis in Mendelian disease with transcriptome sequencing
|Science Translational Medicine|2017|809
Distinctive patterns of microRNA expression in primary muscular disorders
|Proceedings of the National Academy of Sciences|2007|509