title: Induced pluripotent stem cells from ALS patients for disease modeling.
abstract: 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.
authors.affiliation: Johns Hopkins University, School of Medicine, USA.
authors.forename: Jean-Philippe
authors.initials: JP
authors.lastname: Richard
authors.affiliation: Johns Hopkins University, School of Medicine, USA. Electronic address: nmaragak@jhmi.edu.
authors.forename: Nicholas J
authors.initials: NJ
authors.lastname: Maragakis
mesh_terms: D000690:Amyotrophic Lateral Sclerosis / Q000188:drug therapy / Q000503:physiopathology*; D000818:Animals; D004353:Drug Evaluation, Preclinical / Q000191:economics / Q000379:methods; D006801:Humans; D057026:Induced Pluripotent Stem Cells / Q000502:physiology*
keywords: Astrocyte; Human; Motor neuron; Non-cell autonomous; Stem cell; iPSC