Amyotrophic lateral sclerosis: a neurodegenerative disorder poised for successful therapeutic translation

RJ Mead, N Shan, HJ Reiser, F Marshall… - Nature Reviews Drug …, 2023 - nature.com
Amyotrophic lateral sclerosis (ALS) is a devastating disease caused by degeneration of
motor neurons. As with all major neurodegenerative disorders, development of disease …

Amyloid oligomers: A joint experimental/computational perspective on Alzheimer's disease, Parkinson's disease, type II diabetes, and amyotrophic lateral sclerosis

PH Nguyen, A Ramamoorthy, BR Sahoo… - Chemical …, 2021 - ACS Publications
Protein misfolding and aggregation is observed in many amyloidogenic diseases affecting
either the central nervous system or a variety of peripheral tissues. Structural and dynamic …

Organoid-on-a-chip model of human ARPKD reveals mechanosensing pathomechanisms for drug discovery

K Hiratsuka, T Miyoshi, KT Kroll, NR Gupta… - Science …, 2022 - science.org
Organoids serve as a novel tool for disease modeling in three-dimensional multicellular
contexts. Static organoids, however, lack the requisite biophysical microenvironment such …

[HTML][HTML] 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 …

Genetics of amyotrophic lateral sclerosis: seeking therapeutic targets in the era of gene therapy

N Suzuki, A Nishiyama, H Warita, M Aoki - Journal of human genetics, 2023 - nature.com
Amyotrophic lateral sclerosis (ALS) is an intractable disease that causes respiratory failure
leading to mortality. The main locus of ALS is motor neurons. The success of antisense …

In vitro Models of Neurodegenerative Diseases

A Slanzi, G Iannoto, B Rossi, E Zenaro… - Frontiers in cell and …, 2020 - frontiersin.org
Neurodegenerative diseases are progressive degenerative conditions characterized by the
functional deterioration and ultimate loss of neurons. These incurable and debilitating …

[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 …

Glutamatergic dysfunction in Schizophrenia

AO Kruse, JR Bustillo - Translational Psychiatry, 2022 - nature.com
The NMDA-R hypofunction model of schizophrenia started with the clinical observation of
the precipitation of psychotic symptoms in patients with schizophrenia exposed to PCP or …

Mitochondrial bioenergetic deficits in C9orf72 amyotrophic lateral sclerosis motor neurons cause dysfunctional axonal homeostasis

AR Mehta, JM Gregory, O Dando, RN Carter… - Acta …, 2021 - Springer
Axonal dysfunction is a common phenotype in neurodegenerative disorders, including in
amyotrophic lateral sclerosis (ALS), where the key pathological cell-type, the motor neuron …

The role of manganese dysregulation in neurological disease: emerging evidence

D Budinger, S Barral, AKS Soo, MA Kurian - The Lancet Neurology, 2021 - thelancet.com
Manganese is an essential trace metal. The dysregulation of manganese seen in a broad
spectrum of neurological disorders reflects its importance in brain development and key …