Induced pluripotent stem cells from ALS patients for disease modeling

• Induced pluripotent cells have a distinct advantage over embryonic stem cells in modeling ALS.
• IPSC have the capability of being derived from both familial as well as sporadic forms of ALS.
• iPSC can be differentiated into a variety of different ALS-relevant cell subtypes.
• Despite the potential of iPSC, a number of methodological and monetary constraints still exist.
• iPSC have applications in understanding disease mechanisms and screening therapeutics.

The ability to reprogram adult somatic cells into pluripotent stem cells that can differentiate into all three germ layers of the developing human has fundamentally changed the landscape of biomedical research. For a neurodegenerative disease like Amyotrophic Lateral Sclerosis (ALS), which does not manifest itself until adulthood and is a heterogeneous disease with few animal models, this technology may be particularly important. Induced pluripotent stem cells (iPSC) have been created from patients with several familial forms of ALS as well as some sporadic forms of ALS. These cells have been differentiated into ALS-relevant cell subtypes including motor neurons and astrocytes, among others. ALS-relevant pathologies have also been identified in motor neurons from these cells and may provide a window into understanding disease mechanisms in vitro. Given that this is a relatively new field of research, numerous challenges remain before iPSC methodologies can fulfill their potential as tools for modeling ALS as well as providing a platform for the investigation of ALS therapeutics.This article is part of a Special Issue entitled ALS complex pathogenesis.