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Project: PRJNA724722

This study applies targeted Cas9-based gene insertion strategies for the correction of full-length dystrophin in a pre-clinical humanized mouse model of Duchenne muscular dystrophy. Following intramuscular or intravenous delivery, full-length dystrophin is restored in skeletal and cardiac muscle. Overall design: High-throughput unbiased sequencing to characterize and quantify gene-editing outcomes in genomic DNA and cDNA transcripts of treated mice.

Secondary Study Accession:
SRP316076
Study Title:
Full-length dystrophin restoration via targeted genomic integration by AAV-CRISPR in a humanized mouse model of Duchenne muscular dystrophy
Center Name:
Duke University
Study Name:
Full-length dystrophin restoration via targeted genomic integration by AAV-CRISPR in a humanized mouse model of Duchenne muscular dystrophy
ENA-REFSEQ:
N
PROJECT-ID:
724722
ENA-FIRST-PUBLIC:
2021-04-26
ENA-LAST-UPDATE:
2025-02-24

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PRJNA9559 The laboratory mouse is a major model organism for basic mammalian biology, human disease, and genom... See more
The mouse is one of the major organisms for modeling human disease and comparative genome analysis. ... See more
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