Non-invasive Detection of Allele-specific CRISPR-SaCas9-KKH Disruption of TOR1A DYT1 Allele in a Xenograft Mouse Model

Katia E. Maalouf, Dawn Madison Frederick,Nutan Sharma, Edwina Abou Haidar, Tianhe Xiao, Justin Seungkyu Han, Mohammed S. Mahamdeh,Roy J. Soberman,David Rufino-Ramos,Benjamin P. Kleinstiver, Hyder A. Jinnah,Christine A. Vaine,D. Cristopher Bragg,Koen Breyne

Molecular Therapy Nucleic Acids（2025）

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关键词

MT: RNA/DNA Editing,DYT1 dystonia,CRISPR/Cas gene editing,adeno-associated virus vector,extracellular vesicles,theranostic

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