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...

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Detalhes bibliográficos
Publicado no:PLoS Comput Biol
Main Authors: Louie, Wilson, Shen, Max W., Tahiry, Zakir, Zhang, Sophia, Worstell, Daniel, Cassa, Christopher A., Sherwood, Richard I., Gifford, David K.
Formato: Artigo
Idioma:Inglês
Publicado em: Public Library of Science 2021
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Research Article
Acesso em linha: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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