ALS in a dish: development of an in vitro human 3D model of the neuromuscular junction with functional readouts

In amyotrophic lateral sclerosis (ALS), the communication between motor neurons and skeletal muscle is lost, resulting in weakness and degeneration of muscle tissue. ALS animal models fail to reproduce the complexities of the disease, which is thought to account for the failure of new drugs in clinical trials. At The Neuro, human blood samples are obtained to generate pluripotent stem cells (PSCs), which are able to give rise to all the cell types of the human body. Our goal is to generate motor neurons from the PSCs of healthy controls and ALS patients to grow them with skeletal muscle in 3D in a special dish created by eNUVIO. This platorm will facilitate the acquisition of functional readouts similar to those obtained from electromyography and nerve conduction velocity, studies used for ALS diagnosis purposes. Furthermore, the model at the endwill be scalable for high-throughput drug screening purposes to facilitate the identification of lead compounds in our human NMJ on a dish platform.

Faculty Supervisor:

Thomas Durcan

Student:

Maria Castellanos Montiel

Partner:

Enuvio Inc.

Discipline:

Other

Sector:

Manufacturing

University:

Program: