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Series GSE173224 Query DataSets for GSE173224
Status Public on Apr 24, 2021
Title Full-length dystrophin restoration via targeted genomic integration by AAV-CRISPR in a humanized mouse model of Duchenne muscular dystrophy
Organism Mus musculus
Experiment type Other
Summary 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.
Contributor(s) Pickar-Oliver A, Gough V, Bohning JD, Liu S, Robinson-Hamm JN, Daniels H, Majoros WH, Devlin G, Asokan A, Gersbach CA
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Submission date Apr 23, 2021
Last update date Apr 26, 2021
Contact name Adrian Oliver
Phone 3016066963
Organization name Duke University
Street address 101 Science Drive, CIEMAS Rm. 2323
City Durham
State/province North Carolina
ZIP/Postal code 27708
Country USA
Platforms (2)
GPL16417 Illumina MiSeq (Mus musculus)
GPL24247 Illumina NovaSeq 6000 (Mus musculus)
Samples (186)
GSM5262096 PBS_mouse1_local (gDNA)
GSM5262097 PBS_mouse2_local (gDNA)
GSM5262098 g12_1to1_mouse1_local (gDNA)
BioProject PRJNA724722
SRA SRP316076

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SOFT formatted family file(s) SOFTHelp
MINiML formatted family file(s) MINiMLHelp
Series Matrix File(s) TXTHelp

Supplementary file Size Download File type/resource
GSE173224_RAW.tar 525.2 Mb (http)(custom) TAR (of BAM)
GSE173224_cDNA_matrix_editing_counts.txt.gz 598 b (ftp)(http) TXT
GSE173224_gDNA_matrix_editing_counts.txt.gz 1.8 Kb (ftp)(http) TXT
SRA Run SelectorHelp
Raw data are available in SRA
Processed data provided as supplementary file
Processed data are available on Series record

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