5 Steps of Neurodegenerative Disease Modeling

Modeling neurodegenerative disease

Neurodegenerative diseases, such Parkinson’s disease (PD), Alzheimer’s disease (AD), amyotrophic lateral sclerosis (ALS) and Huntington’s disease (HD), affect millions of people worldwide. PD is the fastest growing of these neurological disorders, affects over 6· million people worldwide. Like all neurodegenerative disease which primarily affecting neurons in the human brain, PD is characterized by the selective loss of dopaminergic neurons (DA) in the substantia nigra of the midbrain. Obtaining human cells for in vitro disease models is often a challenge. Parkinson’s and other human disease research has been hindered by a lack of access to diseased tissue to study. It is now possible to develop induced pluripotent stem cells from control or PD patient fibroblasts and differentiate them into neurons.

The pioneering of iPSC technology by Shinya Yamanaka and colleagues presented a very obvious advantage in creating iPSC-based cell models to model human disease. Coupled with advances in gene editing technologies, using iPSCs to build human disease models promises to advance our understanding of disease mechanisms underlying human disease pathologies important to the development of therapeutic drugs and implementation of cell and gene therapies.