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filingDate 2021-04-27-04:00^^<http://www.w3.org/2001/XMLSchema#date>
inventor http://rdf.ncbi.nlm.nih.gov/pubchem/patentinventor/MD5_188b877cb031e0bca95afc062278de94
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publicationDate 2021-11-04-04:00^^<http://www.w3.org/2001/XMLSchema#date>
publicationNumber WO-2021222327-A1
titleOfInvention A high-throughput screening method to discover optimal grna pairs for crispr-mediated exon deletion
abstract Disclosed herein are methods of using probes for high-throughput screening of guide RNA (gRNA) efficiency for Clustered Regularly Interspaced Short Palindromic Repeats (CR!SPR)/CRiSPR-associated (Cas)-based genome editing systems. Further disclosed herein is a humanized transgenic mouse model that recapitulates the severe DMD pathology of human patients. The mouse model may be used for determining the feasibility of CRISPR-based therapies for the correction of the human dystrophin gene by gene editing and methods of use.
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priorityDate 2020-04-27-04:00^^<http://www.w3.org/2001/XMLSchema#date>
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