iPS cells from ALS patients- towards replacing animal models for ALS

ALS is a form of neurodegenerative disorder in which Motor Neurons, the nerves that connect to the muscle die leading loss of movement and affect key body processes such as breathing, eventually causing death. Mouse models for ALS have been generated to test therapeutic interventions but many of these are not robust systems. We propose to make stem cell like cells from skin cells of ALS patients from which we can make Motor neurons. These motor neurons will provide a model more akin to the human disease and therefore serve a good model to understand the disease process as well as to test drugs.

Animal models of diseases have been used extensively to study the disease process as as well as to test therapeutic interventions. However, in many cases these rodent models do not provide a system which mimic the human disease completely. Moreover it is important to reduce the use of animals in scientific research and use equally robust invitro culture system.
We propose to derive patient specific induced pluripotent stem cells from patients with Amyotrophic lateral sclerosis (ALS). We will use dermal fibroblast cells from patients who have ALS (caused by mutations in the Angiogenin gene) to derive induced pluripotent stem cells. We will then induce these iPS cells to differentiate into Motor neurons and study their cellular and molecular phenotype to understand Ang mediated ALS. This will provide a robust model system to study ALS and replace animal models for Ang mediated ALS.