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Amyotrophic lateral sclerosis: a neurodegenerative disorder poised for successful therapeutic translation
Amyotrophic lateral sclerosis (ALS) is a devastating disease caused by degeneration of
motor neurons. As with all major neurodegenerative disorders, development of disease …
motor neurons. As with all major neurodegenerative disorders, development of disease …
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) …
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 …
TDP-43 represses cryptic exon inclusion in the FTD–ALS gene UNC13A
A hallmark pathological feature of the neurodegenerative diseases amyotrophic lateral
sclerosis (ALS) and frontotemporal dementia (FTD) is the depletion of RNA-binding protein …
sclerosis (ALS) and frontotemporal dementia (FTD) is the depletion of RNA-binding protein …
Mis-spliced transcripts generate de novo proteins in TDP-43–related ALS/FTD
Functional loss of TDP-43, an RNA binding protein genetically and pathologically linked to
amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD), leads to the …
amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD), leads to the …
Neuropathogenesis-on-chips for neurodegenerative diseases
S Amartumur, H Nguyen, T Huynh, TS Kim… - Nature …, 2024 - nature.com
Develo** diagnostics and treatments for neurodegenerative diseases (NDs) is
challenging due to multifactorial pathogenesis that progresses gradually. Advanced in vitro …
challenging due to multifactorial pathogenesis that progresses gradually. Advanced in vitro …
A fluid biomarker reveals loss of TDP-43 splicing repression in presymptomatic ALS–FTD
Although loss of TAR DNA-binding protein 43 kDa (TDP-43) splicing repression is well
documented in postmortem tissues of amyotrophic lateral sclerosis (ALS) and frontotemporal …
documented in postmortem tissues of amyotrophic lateral sclerosis (ALS) and frontotemporal …
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 …
Splicing regulation through biomolecular condensates and membraneless organelles
Biomolecular condensates, sometimes also known as membraneless organelles (MLOs),
can form through weak multivalent intermolecular interactions of proteins and nucleic acids …
can form through weak multivalent intermolecular interactions of proteins and nucleic acids …
Advances in molecular pathology, diagnosis, and treatment of amyotrophic lateral sclerosis
H Ilieva, M Vullaganti, J Kwan - bmj, 2023 - bmj.com
Although the past two decades have produced exciting discoveries in the genetics and
pathology of amyotrophic lateral sclerosis (ALS), progress in develo** an effective therapy …
pathology of amyotrophic lateral sclerosis (ALS), progress in develo** an effective therapy …