Drug screening for ALS using patient-specific induced pluripotent stem cells

N Egawa, S Kitaoka, K Tsukita, M Naitoh… - Science translational …, 2012 - science.org
Amyotrophic lateral sclerosis (ALS) is a late-onset, fatal disorder in which the motor neurons
degenerate. The discovery of new drugs for treating ALS has been hampered by a lack of …

A cellular model for sporadic ALS using patient-derived induced pluripotent stem cells

MF Burkhardt, FJ Martinez, S Wright, C Ramos… - Molecular and Cellular …, 2013 - Elsevier
Abstract Development of therapeutics for genetically complex neurodegenerative diseases
such as sporadic amyotrophic lateral sclerosis (ALS) has largely been hampered by lack of …

Induced pluripotent stem cells from ALS patients for disease modeling

JP Richard, NJ Maragakis - Brain research, 2015 - Elsevier
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 …

[HTML][HTML] Induced pluripotent stem cells and their applications in amyotrophic lateral sclerosis

H Du, Z Huo, Y Chen, Z Zhao, F Meng, X Wang, S Liu… - Cells, 2023 - mdpi.com
Amyotrophic lateral sclerosis (ALS) is a progressive neurodegenerative disease that results
in the loss of motor function in the central nervous system (CNS) and ultimately death. The …

Distinct responses of neurons and astrocytes to TDP-43 proteinopathy in amyotrophic lateral sclerosis

P Smethurst, E Risse, GE Tyzack, JS Mitchell, DM Taha… - Brain, 2020 - academic.oup.com
Amyotrophic lateral sclerosis (ALS) is a fatal and incurable neurodegenerative disease
caused by motor neuron loss, resulting in muscle wasting, paralysis and eventual death. A …

Modeling ALS with motor neurons derived from human induced pluripotent stem cells

S Sances, LI Bruijn, S Chandran, K Eggan, R Ho… - Nature …, 2016 - nature.com
Directing the differentiation of induced pluripotent stem cells into motor neurons has allowed
investigators to develop new models of amyotrophic lateral sclerosis (ALS). However …

[HTML][HTML] Large-scale differentiation of iPSC-derived motor neurons from ALS and control subjects

MJ Workman, RG Lim, J Wu, A Frank, L Ornelas… - Neuron, 2023 - cell.com
Using induced pluripotent stem cells (iPSCs) to understand the mechanisms of neurological
disease holds great promise; however, there is a lack of well-curated lines from a large array …

[HTML][HTML] Spinal cord extracts of amyotrophic lateral sclerosis spread TDP-43 pathology in cerebral organoids

Y Tamaki, JP Ross, P Alipour, CÉ Castonguay… - PLoS …, 2023 - journals.plos.org
Amyotrophic lateral sclerosis (ALS) is a fatal neurodegenerative disorder caused by
progressive loss of motor neurons and there is currently no effective therapy. Cytoplasmic …

4-Aminopyridine induced activity rescues hypoexcitable motor neurons from amyotrophic lateral sclerosis patient-derived induced pluripotent stem cells

M Naujock, N Stanslowsky, S Bufler, M Naumann… - Stem Cells, 2016 - academic.oup.com
Despite decades of research on amyotrophic lateral sclerosis (ALS), there is only one
approved drug, which minimally extends patient survival. Here, we investigated …

[HTML][HTML] Human iPSC-derived motoneurons harbouring TARDBP or C9ORF72 ALS mutations are dysfunctional despite maintaining viability

AC Devlin, K Burr, S Borooah, JD Foster… - Nature …, 2015 - nature.com
Amyotrophic lateral sclerosis (ALS) is a devastating neurodegenerative disease for which a
greater understanding of early disease mechanisms is needed to reveal novel therapeutic …