[PDF][PDF] iPSC-based disease modeling and drug discovery in cardinal neurodegenerative disorders

H Okano, S Morimoto - Cell Stem Cell, 2022 - cell.com
It has been 15 years since the birth of human induced pluripotent stem cell (iPSC)
technology in 2007, and the scope of its application has been expanding. In addition to 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 …

[HTML][HTML] Integrated transcriptome landscape of ALS identifies genome instability linked to TDP-43 pathology

OJ Ziff, J Neeves, J Mitchell, G Tyzack… - Nature …, 2023 - nature.com
Abstract Amyotrophic Lateral Sclerosis (ALS) causes motor neuron degeneration, with 97%
of cases exhibiting TDP-43 proteinopathy. Elucidating pathomechanisms has been …

[HTML][HTML] Identification of therapeutic targets for amyotrophic lateral sclerosis using PandaOmics–an AI-enabled biological target discovery platform

FW Pun, BHM Liu, X Long, HW Leung… - Frontiers in Aging …, 2022 - frontiersin.org
Amyotrophic lateral sclerosis (ALS) is a severe neurodegenerative disease with ill-defined
pathogenesis, calling for urgent developments of new therapeutic regimens. Herein, we …

[HTML][HTML] Answer ALS, a large-scale resource for sporadic and familial ALS combining clinical and multi-omics data from induced pluripotent cell lines

EG Baxi, T Thompson, J Li, JA Kaye, RG Lim… - Nature …, 2022 - nature.com
Answer ALS is a biological and clinical resource of patient-derived, induced pluripotent stem
(iPS) cell lines, multi-omic data derived from iPS neurons and longitudinal clinical and …

[HTML][HTML] Functional genomics in stroke: current and future applications of iPSCs and gene editing to dissect the function of risk variants

A Granata - BMC cardiovascular disorders, 2023 - Springer
Stroke is an important disease with unmet clinical need. To uncover novel paths for
treatment, it is of critical importance to develop relevant laboratory models that may help to …

[HTML][HTML] Loss of PML nuclear bodies in familial amyotrophic lateral sclerosis-frontotemporal dementia

F Antoniani, M Cimino, L Mediani, J Vinet… - Cell death …, 2023 - nature.com
Abstract Amyotrophic Lateral Sclerosis (ALS) and Frontotemporal Dementia (FTD) are two
neurodegenerative disorders that share genetic causes and pathogenic mechanisms. The …

[HTML][HTML] Induced pluripotent stem cell-based drug screening by use of artificial intelligence

D Kusumoto, S Yuasa, K Fukuda - Pharmaceuticals, 2022 - mdpi.com
Induced pluripotent stem cells (iPSCs) are terminally differentiated somatic cells that
differentiate into various cell types. iPSCs are expected to be used for disease modeling and …

[HTML][HTML] The role of extracellular matrix components in the spreading of pathological protein aggregates

E Moretto, S Stuart, S Surana, JNS Vargas… - Frontiers in Cellular …, 2022 - frontiersin.org
Several neurodegenerative diseases are characterized by the accumulation of aggregated
misfolded proteins. These pathological agents have been suggested to propagate in the …

[HTML][HTML] PolyGR and polyPR knock-in mice reveal a conserved neuroprotective extracellular matrix signature in C9orf72 ALS/FTD neurons

C Milioto, M Carcolé, A Giblin, R Coneys… - Nature …, 2024 - nature.com
Dipeptide repeat proteins are a major pathogenic feature of C9orf72 amyotrophic lateral
sclerosis (C9ALS)/frontotemporal dementia (FTD) pathology, but their physiological impact …