Machine learning based CRISPR gRNA design for therapeutic exon skipping
Machine learning based CRISPR gRNA design for therapeutic exon skipping
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United States: Public Library of Science
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Language
English
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Publisher
United States: Public Library of Science
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Contents
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 genome editing that causes exon skipping is a promis...
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Full title
Machine learning based CRISPR gRNA design for therapeutic exon skipping
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TN_cdi_plos_journals_2490328187
Permalink
https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_plos_journals_2490328187
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ISSN
1553-7358,1553-734X
E-ISSN
1553-7358
DOI
10.1371/journal.pcbi.1008605
