Alec Smith has been awarded a two-year R03 grant from NIH entitled Using functional readouts from engineering models of innervated skeletal muscle to assess the efficacy of CRISPR-based c9orf72 ALS gene therapies.
This project is part of a larger effort to employ human induced pluripotent stem cell-derived motor neurons and skeletal muscle to produce in vitro models of the neuromuscular junction to model motor neuron diseases in order to study disease mechanisms and for drug screens.