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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Bibliografske podrobnosti
izdano v:Sci Transl Med
Main Authors: 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
Jezik:Inglês
Izdano: 2020
Teme:
Article
Online dostop: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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