Orthopedic & Muscular System: Current Research
Open Access

Establishment of a human-based in vitro functional NMJ system for ALS drug screening

International Conference and Expo on Biomechanics & Implant Design

July 27-29, 2015 Orlando, USA

Xiufang Guo, Yunqing Cai, Navaneetha Santhanam, Christopher McAleer, Balaji Srinivasan Venkatesh, Ying Wang, Frank Sommerhage, Candace Martin and James Hickman

Posters-Accepted Abstracts: Orthop Muscular Syst

Abstract :

The neuromuscular system represents the major functional outputs of the central nervous system (CNS). Its dysfunction is closely related to many neurodegenerative diseases, including ALS (Amyotrophic Lateral Sclerosis). This study aims to develop a defined human-based functional neuromuscular system for the study and drug development of ALS disease. We have developed a defined in vitro system in which functional neuromuscular junctions (NMJs) can be formed with human stem cells as the sources. With the implementation of PDMS chamber system, this NMJ system is then modified by separating motoneurons and muscle cells, in which the functional analysis of NMJ formation can be better controlled. Motoneurons and muscle cells will be differentiated from the iPSCs derived from diseased patients. These patient-derived cells will then be incorporated into this functional NMJ system for the pathological and therapeutic study of disease. The outcome of this study would generate efficient functional in vitro model which would be clinically applicable to the study and treatment of various NMJ pathologies..