May 6, 2025

Elaine Lai (PhD student, Eguchi lab) and Lan Weiss, MD, PhD (Project Scientist, Kimonis lab) have been awarded the MAXIMUS Seed Grant on “Optimizing Muscle-Specific Promoters in iPSC-Derived Myotubes for VCP Disease Gene Therapy.” This seed grant provides $5,000 for a collaborative project between two labs. The goal of this study is to compare various promoters currently in preclinical and clinical trials in human iPSC-myotubes bearing a missense mutation in Valosin-Containing Protein (VCP). While these selected promoters have shown promising results in mouse models, their efficacy in human cells with disease-causing mutations are unknown. This collaborative project will generate data that will guide the design of gene therapies for VCP.