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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 …

Therapy Q&A

Interview of Dr. Joel Chamberlain regarding her lab's work on therapy development.
Thanks to Dr. Joel Chamberlain (University of Washington, Seattle) who answered our questions about the work that she and her lab are doing to better understand FSHD and develop su…