Specific DMPK-Promoter Targeting by CRISPRi Reverses Myotonic Dystrophy Type 1-Associated Defects in Patient Muscle Cells

The authors investigated a DMPK-promoter silencing strategy using CRISPR interference as a new alternative approach. Different sgRNAs targeting the DMPK promoter were evaluated in m yotonic dystrophy type 1 patient muscle cells.
[Molecular Therapy – Nucleic Acids]
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