Amyotrophic lateral sclerosis: translating genetic discoveries into therapies
Recent advances in sequencing technologies and collaborative efforts have led to
substantial progress in identifying the genetic causes of amyotrophic lateral sclerosis (ALS) …
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 …
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
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 …
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 …
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
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 …
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 …
throughout the central nervous system (CNS). By modulating RNA, they hold the promise of …
Clonally expanded CD8 T cells characterize amyotrophic lateral sclerosis-4
Amyotrophic lateral sclerosis (ALS) is a heterogenous neurodegenerative disorder that
affects motor neurons and voluntary muscle control. ALS heterogeneity includes the age of …
affects motor neurons and voluntary muscle control. ALS heterogeneity includes the age of …
[HTML][HTML] Antisense Oligonucleotides for the Study and Treatment of ALS
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 …
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 …
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 …
In the past 2 years, the understanding of the downstream mechanisms of several ALS genes …