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) …

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 …

Mechanism of STMN2 cryptic splice-polyadenylation and its correction for TDP-43 proteinopathies

MW Baughn, Z Melamed, J López-Erauskin… - Science, 2023 - science.org
Loss of nuclear TDP-43 is a hallmark of neurodegeneration in TDP-43 proteinopathies,
including amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD). TDP-43 …

Current state and future directions in the therapy of ALS

L Tzeplaeff, S Wilfling, MV Requardt, M Herdick - Cells, 2023 - mdpi.com
Amyotrophic lateral sclerosis (ALS) is a rapidly progressive neurodegenerative disorder
affecting upper and lower motor neurons, with death resulting mainly from respiratory failure …

Pharmacotherapy for amyotrophic lateral sclerosis: a review of approved and upcoming agents

SA Johnson, T Fang, F De Marchi, D Neel… - Drugs, 2022 - Springer
Amyotrophic lateral sclerosis (ALS) is a rapidly progressive neurodegenerative disorder
involving loss of upper and lower motor neurons, with most cases ending in death within 3–5 …

A single-cell map of antisense oligonucleotide activity in the brain

MA Mortberg, JE Gentile, NM Nadaf… - Nucleic Acids …, 2023 - academic.oup.com
Antisense oligonucleotides (ASOs) dosed into cerebrospinal fluid (CSF) distribute broadly
throughout the central nervous system (CNS). By modulating RNA, they hold the promise of …

Clonally expanded CD8 T cells characterize amyotrophic lateral sclerosis-4

L Campisi, S Chizari, JSY Ho, A Gromova, FJ Arnold… - Nature, 2022 - nature.com
Amyotrophic lateral sclerosis (ALS) is a heterogenous neurodegenerative disorder that
affects motor neurons and voluntary muscle control. ALS heterogeneity includes the age of …

[HTML][HTML] Antisense Oligonucleotides for the Study and Treatment of ALS

BD Boros, KM Schoch, CJ Kreple, TM Miller - Neurotherapeutics, 2022 - Elsevier
Amyotrophic lateral sclerosis (ALS) is a fatal neurodegenerative disease characterized by
motor neuron loss. ALS is now associated with mutations in numerous genes, many of which …

New developments and opportunities in drugs being trialed for amyotrophic lateral sclerosis from 2020 to 2022

JS Jiang, Y Wang, M Deng - Frontiers in pharmacology, 2022 - frontiersin.org
Amyotrophic lateral sclerosis (ALS) is a progressive neurodegenerative disorder that
primarily affects motor neurons in the brain and spinal cord. In the recent past, there have …

Update on genetics of amyotrophic lateral sclerosis

D Brenner, A Freischmidt - Current Opinion in Neurology, 2022 - journals.lww.com
The genetic and molecular basis of ALS is increasingly examined on single-cell resolution.
In the past 2 years, the understanding of the downstream mechanisms of several ALS genes …