Titolo: MiMiC Model Muscle Construct: a 3D organoid mimicking native skeletal muscle as a tool for testing the efficacy of new pharmacological therapeutic molecules

Bando: PRIN PNRR

Durata: 24 mesi

Coordinatore: PROF.SSA ROBERTA SACCHETTO

Budget BCA: 136.985 EU

Responsabile scientifico: prof.ssa Roberta Sacchetto

Research Team: Sacchetto Roberta

Abstract: The main aim of the project is the development and validation of cheap and easy to obtain 3D muscle models based on two different scaffolds, namely de-cellularized extracellular matrix from bovine diaphragm and collagen-based scaffold derived from non-processed sea urchin food waste, both re-cellularized with primary myoblasts. The as-obtained 3D models will be used as a platform for the development of innovative therapeutic strategies for neuromuscular disorders and can be employed in parallel with conventional in vitro studies and as alternative of in vivo animal models. In particular, we intend to generate tissue models with very high proximity to muscle physiology to validate the efficacy of pharmacological molecules already selected in vitro to cure human Brody disease (BD), a rare pathology affecting voluntary muscles.
The research team possesses a long experience in studying rare inherited neuromuscular diseases both in cattle (Congenital Pseudomyotonia and KCNG1 neuromuscular channelopathy) and human species (BD and Limb-girdle muscular dystrophy) as well as in developing biomimetic scaffolds for biomedical use.
The main goal of the project is to provide robust, biologically relevant and reproducible muscle tissue models to be strategically exploited in pre-clinical studies. The project will be divided into 3 milestones:
1) generation, refinement and characterization of 3D muscle construct made by decellularized extracellular matrix from bovine diaphragm
2) generation and characterization of 3D muscle constructs using sea urchin collagen as biomimetic scaffold
3) use of the as-obtained 3D muscle constructs for testing efficacy and safety of pharmaceutical compounds known as CFTR correctors, proposed to treat Brody disease