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Long-term evaluation of AAV-CRISPR genome editing for Duchenne muscular dystrophy.
Nat Med. 2019 Mar;25(3):427-432. doi: 10.1038/s41591-019-0344-3. Epub 2019 Feb 18.
Nat Med. 2019.
PMID: 30778238
Free PMC article.
Full-length dystrophin restoration via targeted exon integration by AAV-CRISPR in a humanized mouse model of Duchenne muscular dystrophy.
Pickar-Oliver A, Gough V, Bohning JD, Liu S, Robinson-Hamm JN, Daniels H, Majoros WH, Devlin G, Asokan A, Gersbach CA.
Pickar-Oliver A, et al. Among authors: bohning jd.
Mol Ther. 2021 Nov 3;29(11):3243-3257. doi: 10.1016/j.ymthe.2021.09.003. Epub 2021 Sep 10.
Mol Ther. 2021.
PMID: 34509668
Free PMC article.
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Myogenic Progenitor Cell Lineage Specification by CRISPR/Cas9-Based Transcriptional Activators.
Kwon JB, Vankara A, Ettyreddy AR, Bohning JD, Gersbach CA.
Kwon JB, et al. Among authors: bohning jd.
Stem Cell Reports. 2020 May 12;14(5):755-769. doi: 10.1016/j.stemcr.2020.03.026. Epub 2020 Apr 23.
Stem Cell Reports. 2020.
PMID: 32330446
Free PMC article.
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In Vivo Gene Editing of Muscle Stem Cells with Adeno-Associated Viral Vectors in a Mouse Model of Duchenne Muscular Dystrophy.
Kwon JB, Ettyreddy AR, Vankara A, Bohning JD, Devlin G, Hauschka SD, Asokan A, Gersbach CA.
Kwon JB, et al. Among authors: bohning jd.
Mol Ther Methods Clin Dev. 2020 Sep 28;19:320-329. doi: 10.1016/j.omtm.2020.09.016. eCollection 2020 Dec 11.
Mol Ther Methods Clin Dev. 2020.
PMID: 33145368
Free PMC article.
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