A perspective on stem cell modeling of amyotrophic lateral sclerosis.
Amyotrophic lateral sclerosis is a complex neurodegenerative disease. Limitations in animal models have impeded progress in studying disease pathology and potential drug discovery. Here, we will review recent advances in the development of stem cell models for the study of ALS. Additionally, we will discuss the progress toward therapeutic development derived from these stem cell based assays.
a The Howard Hughes Medical Institute; The Harvard Stem Cell Institute; The Stanley Center for Psychiatric Research ; The Department of Stem Cell and Regenerative Biology ; Harvard University ; Cambridge , MA USA.|b Department of Anatomy & Embryology ; Leiden University Medical Center ; The Netherlands.
A Sophie
AS
de Boer
a The Howard Hughes Medical Institute; The Harvard Stem Cell Institute; The Stanley Center for Psychiatric Research ; The Department of Stem Cell and Regenerative Biology ; Harvard University ; Cambridge , MA USA.
Kevin
K
Eggan
D000690:Amyotrophic Lateral Sclerosis / Q000235:genetics / Q000473:pathology*; D001253:Astrocytes / Q000502:physiology; D018929:Cell Culture Techniques; D002454:Cell Differentiation; D004268:DNA-Binding Proteins / Q000235:genetics; D006801:Humans; D057026:Induced Pluripotent Stem Cells; D017628:Microglia / Q000502:physiology; D034702:RNA-Binding Protein FUS / Q000235:genetics; D013482:Superoxide Dismutase / Q000235:genetics; D000072105:Superoxide Dismutase-1; D033921:Vesicular Transport Proteins / Q000235:genetics
D004268:DNA-Binding Proteins; C000599550:FUS protein, human; D034702:RNA-Binding Protein FUS; C000606290:SOD1 protein, human; C000598624:TARDBP protein, human; C496748:VAPB protein, human; D033921:Vesicular Transport Proteins; D013482:Superoxide Dismutase; D000072105:Superoxide Dismutase-1
Amyotrophic Lateral Sclerosis; astrocyte; embryonic stem cells; glia; induced pluripotent stem cells; microglia; motor neuron