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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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Bibliografiska uppgifter
I publikationen:Sci Transl Med
Huvudupphovsmän: 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.
Materialtyp: Artigo
Språk:Inglês
Publicerad: 2020
Ämnen:
Länkar: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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