AUF1 gene therapy

AUF1 gene therapy is an experimental gene therapy being developed by REGENXBIO in collaboration with New York University for the treatment of various muscular dystrophies and muscle wasting conditions. The therapy utilizes an adeno-associated virus serotype 8 (AAV8) vector to deliver the AUF1 (AU-rich element RNA-binding factor 1, also known as HNRNPD) gene directly to skeletal muscle tissue. AUF1 is a critical RNA-binding protein that regulates the stability and translation of numerous mRNAs involved in muscle development and maintenance. In conditions like Duchenne muscular dystrophy (DMD), the therapy works by upregulating utrophin, a functional homolog of dystrophin, while also promoting muscle stem cell (satellite cell) activation and improving mitochondrial biogenesis. Preclinical studies have demonstrated its potential to reduce muscle atrophy, accelerate regeneration, and improve overall muscle strength and exercise performance in models of DMD, limb-girdle muscular dystrophy (LGMD), sarcopenia, and traumatic muscle injury.

Other names

HNRNPD gene therapyAAV8-AUF1AAV-8-AUF1AAV 8-AUF1