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.
Organism:
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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Study Accession | Sample Accession | Experiment Accession | Run Accession | Tax Id | Scientific Name | ||||
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Mus musculus | N/ASubmitted files are only available for data submitted via ENA | N/A | |||||||
Mus musculus | N/ASubmitted files are only available for data submitted via ENA | N/A | |||||||
Mus musculus | N/ASubmitted files are only available for data submitted via ENA | N/A | |||||||
Mus musculus | N/ASubmitted files are only available for data submitted via ENA | N/A | |||||||
Mus musculus | N/ASubmitted files are only available for data submitted via ENA | N/A | |||||||
Mus musculus | N/ASubmitted files are only available for data submitted via ENA | N/A | |||||||
Mus musculus | N/ASubmitted files are only available for data submitted via ENA | N/A | |||||||
Mus musculus | N/ASubmitted files are only available for data submitted via ENA | N/A | |||||||
Mus musculus | N/ASubmitted files are only available for data submitted via ENA | N/A | |||||||
Mus musculus | N/ASubmitted files are only available for data submitted via ENA | N/A |
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