AAV1-Follistatin / Milo Biotechnology - LARVOL DELTA

AAV-mediated follistatin gene therapy improves functional outcomes in the TIC-DUX4 mouse model of FSHD. (PubMed, JCI Insight) - "Here, we report such a model - the tamoxifen-inducible FSHD mouse model called TIC-DUX4. AAV1.Follistatin significantly increased TIC-DUX4 muscle mass and strength even in the presence of DUX4 expression, suggesting that myostatin inhibition may be a promising approach to treat FSHD-associated weakness. We conclude that TIC-DUX4 mice are a relevant model to study DUX4 toxicity and, importantly, are useful in therapeutic development studies for FSHD."

Journal • Preclinical