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

Posted by George Shaw on January 17, 2018

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 was increased to clinically relevant doses. The authors express confidence in the potential of AAV-delivered mi405 as a clinical trial candidate.

See original article.

This research was partially funded by Friends through the grant Validation of a PPMO morpholino lead compound in an animal model of FSHD.