Applying genome-wide CRISPR-Cas9 screens for therapeutic discovery in facioscapulohumeral muscular dystrophy

The emergence of CRISPR-Cas9 gene-editing technologies and genome-wide CRISPR-Cas9 libraries enables efficient unbiased genetic screening that can accelerate the process of therapeutic discovery for genetic disorders. Here, we demonstrate the utility of a genome-wide CRISPR-Cas9 loss-of-function lib...

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Publicat a:Sci Transl Med
Autors principals: Lek, Angela, Zhang, Yuanfan, Woodman, Keryn G., Huang, Shushu, DeSimone, Alec M., Cohen, Justin, Ho, Vincent, Conner, James, Mead, Lillian, Kodani, Andrew, Pakula, Anna, Sanjana, Neville, King, Oliver D., Jones, Peter L., Wagner, Kathryn R., Lek, Monkol, Kunkel, Louis M.
Format: Artigo
Idioma:Inglês
Publicat: 2020
Matèries:
Article
Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC7304480/
https://ncbi.nlm.nih.gov/pubmed/32213627
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1126/scitranslmed.aay0271
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