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Pre-clinical Safety and Off-Target Studies to Support Translation of AAV-Mediated RNAi Therapy for FSHD

A publication from Scott Harper's lab reporting on the use of an AAV vector to test the safety of two miRNAs in silencing DUX4.
Harper's lab had previously designed two miRNAs, labeled mi1155 and mi405, and.had performed a low dosage test on mouse muscle. This paper describes what happened when the dosage …