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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.