Award
NIH Reporter #5R01AR082979-04
Engineering a Human Skeletal Muscle Tissue Model of LGMD2B
Recipient
Duke University, Durham, NC
Award Amount
$507,665.00
Ceiling
$507,665.00
Awarded
July 07, 2026
Identifier
5R01AR082979-04
Development of a 3D human skeletal muscle tissue model to study LGMD2B disease mechanisms, fat formation, inflammation, and cholesterol metabolism, using human iPSC lines and patient biopsies, with the goal of facilitating clinical trials.
Description
This project aims to develop a novel 3D human skeletal muscle tissue model replicating the main structural, functional, and metabolic features of LGMD2B using human iPSC lines from healthy and LGMD2B donors. It includes developing a tissue-engineered model of intramuscular adipose tissue accumulation, studying heterocellular interactions and inflammatory responses, and investigating impaired cholesterol metabolism in LGMD2B muscle. The model aims to enable mechanistic and pharmacological studies leading to clinical trials for LGMD2B.