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...

Descripción completa

Detalles Bibliográficos
Autores principales: Louie, Wilson, Shen, Max W., Tahiry, Zakir, Zhang, Sophia, Worstell, Daniel, Cassa, Christopher A., Sherwood, Richard I., Gifford, David K.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Public Library of Science 2021
Materias:
Research Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7819613/
https://www.ncbi.nlm.nih.gov/pubmed/33417623
http://dx.doi.org/10.1371/journal.pcbi.1008605

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7819613/
https://www.ncbi.nlm.nih.gov/pubmed/33417623
http://dx.doi.org/10.1371/journal.pcbi.1008605

Ejemplares similares