Grant 48


Research Institution: Hubrecht Institute

Principle Investigator:

Professor Niels Geijsen

Type: International - Utrecht, Netherlands

Project title: Muscle-in-a-dish, development of an in vitro platform of human skeletal muscle

Status: Active

Summary

FSHD is a human-specific disease and current in vitro and in vivo models do not fully recapitulate the genetic and pathophysiologic aspects of the disease. Therefore, the rationale of this project is to create a 3-D in-vitro FSHD muscle model that mimics the human pathophysiology of FSHD as close as possible. The ability to grow skeletal muscle in a 3D environment coupled with electrostimulation of the muscle tissue allows improved maturation of the in vitro-derived skeletal muscle tissue and provides a new platform on which to test functional parameters of human muscle physiology and test new therapeutic approaches to treat FSHD. In this project, we aim to generate human 3D muscle tissue in vitro to model the natural physiological state and pathogenesis of FSHD, and enable the assessment of the efficacy of different gene editing approaches for the treatment of this disease.