Machine learning based CRISPR gRNA design for therapeutic exon skipping

Restoring gene function by the induced skipping of deleterious exons has been shown to be effective for treating genetic disorders. However, many of the clinically successful therapies for exon skipping are transient oligonucleotide-based treatments that require frequent dosing. CRISPR-Cas9 based ge...

Disgrifiad llawn

Wedi'i Gadw mewn:
Manylion Llyfryddiaeth
Cyhoeddwyd yn:PLoS Comput Biol
Prif Awduron: Louie, Wilson, Shen, Max W., Tahiry, Zakir, Zhang, Sophia, Worstell, Daniel, Cassa, Christopher A., Sherwood, Richard I., Gifford, David K.
Fformat: Artigo
Iaith:Inglês
Cyhoeddwyd: Public Library of Science 2021
Pynciau:
Research Article
Mynediad Ar-lein:https://ncbi.nlm.nih.gov/pmc/articles/PMC7819613/
https://ncbi.nlm.nih.gov/pubmed/33417623
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1371/journal.pcbi.1008605
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