Associated Papers
Specific DMPK-promoter targeting by CRISPRi reverses myotonic dystrophy type 1-associated defects in patient muscle cells.
Porquet F et al.
Molecular Therapy. Nucleic Acids, 2023
Comprehensive analysis and accurate quantification of unintended large gene modifications induced by CRISPR-Cas9 gene editing.
Park SH et al.
Science Advances, 2022
Unexpected Mutations by CRISPR-Cas9 CTG Repeat Excision in Myotonic Dystrophy and Use of CRISPR Interference as an Alternative Approach.
Ikeda M et al.
Molecular Therapy. Methods & Clinical Development, 2020
Genome Editing of Expanded CTG Repeats within the Human DMPK Gene Reduces Nuclear RNA Foci in the Muscle of DM1 Mice.
Lo Scrudato M et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy, 2019
Myoediting: Toward Prevention of Muscular Dystrophy by Therapeutic Genome Editing.
Zhang Y et al.
Physiological Reviews, 2018
Impeding Transcription of Expanded Microsatellite Repeats by Deactivated Cas9.
Pinto BS et al.
Molecular Cell, 2017
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