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 …

Amyotrophic lateral sclerosis: translating genetic discoveries into therapies

F Akçimen, ER Lopez, JE Landers, A Nath… - Nature Reviews …, 2023 - nature.com
Recent advances in sequencing technologies and collaborative efforts have led to
substantial progress in identifying the genetic causes of amyotrophic lateral sclerosis (ALS) …

Context-dependent and disease-specific diversity in protein interactions within stress granules

S Markmiller, S Soltanieh, KL Server, R Mak, W Jin… - Cell, 2018 - cell.com
Stress granules (SGs) are transient ribonucleoprotein (RNP) aggregates that form during
cellular stress and are increasingly implicated in human neurodegeneration. To study the …

[HTML][HTML] RNA granules hitchhike on lysosomes for long-distance transport, using annexin A11 as a molecular tether

YC Liao, MS Fernandopulle, G Wang, H Choi, L Hao… - Cell, 2019 - cell.com
Long-distance RNA transport enables local protein synthesis at metabolically-active sites
distant from the nucleus. This process ensures an appropriate spatial organization of …

Emerging aqueous two-phase systems: from fundamentals of interfaces to biomedical applications

Y Chao, HC Shum - Chemical Society Reviews, 2020 - pubs.rsc.org
Aqueous two-phase systems (ATPSs) have been recognized for their applications in
extraction, separation, purification, and enrichment of (bio) molecules and cells. Recently …

Disruption of axonal transport in neurodegeneration

SH Berth, TE Lloyd - The Journal of Clinical Investigation, 2023 - Am Soc Clin Investig
Neurons are markedly compartmentalized, which makes them reliant on axonal transport to
maintain their health. Axonal transport is important for anterograde delivery of newly …

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 …

ALS genes in the genomic era and their implications for FTD

HP Nguyen, C Van Broeckhoven, J van der Zee - Trends in Genetics, 2018 - cell.com
Amyotrophic lateral sclerosis (ALS) is a complex neurodegenerative disease, characterized
genetically by a disproportionately large contribution of rare genetic variation. Driven by …

RNA transport and local translation in neurodevelopmental and neurodegenerative disease

MS Fernandopulle, J Lippincott-Schwartz… - Nature …, 2021 - nature.com
Neurons decentralize protein synthesis from the cell body to support the active metabolism
of remote dendritic and axonal compartments. The neuronal RNA transport apparatus …

Single-cell transcriptomic landscape of the developing human spinal cord

J Andersen, N Thom, JL Shadrach, X Chen… - Nature …, 2023 - nature.com
Understanding spinal cord assembly is essential to elucidate how motor behavior is
controlled and how disorders arise. The human spinal cord is exquisitely organized, and this …