Molecular mechanisms of TDP-43 misfolding and pathology in amyotrophic lateral sclerosis

A Prasad, V Bharathi, V Sivalingam… - Frontiers in molecular …, 2019 - frontiersin.org
TAR DNA binding protein 43 (TDP-43) is a versatile RNA/DNA binding protein involved in
RNA-related metabolism. Hyper-phosphorylated and ubiquitinated TDP-43 deposits act as …

Alternative splicing as a regulator of development and tissue identity

FE Baralle, J Giudice - Nature reviews Molecular cell biology, 2017 - nature.com
Alternative splicing of eukaryotic transcripts is a mechanism that enables cells to generate
vast protein diversity from a limited number of genes. The mechanisms and outcomes of …

[HTML][HTML] RNA binding antagonizes neurotoxic phase transitions of TDP-43

JR Mann, AM Gleixner, JC Mauna, E Gomes… - Neuron, 2019 - cell.com
TDP-43 proteinopathy is a pathological hallmark of amyotrophic lateral sclerosis and
frontotemporal dementia where cytoplasmic TDP-43 inclusions are observed within …

Gene therapy for neurological disorders: progress and prospects

BE Deverman, BM Ravina, KS Bankiewicz… - Nature Reviews Drug …, 2018 - nature.com
Adeno-associated viral (AAV) vectors are a rapidly emerging gene therapy platform for the
treatment of neurological diseases. In preclinical studies, transgenes encoding therapeutic …

The role of TDP-43 propagation in neurodegenerative diseases: integrating insights from clinical and experimental studies

M Jo, S Lee, YM Jeon, S Kim, Y Kwon… - Experimental & molecular …, 2020 - nature.com
Abstract TAR DNA-binding protein 43 (TDP-43) is a highly conserved nuclear RNA/DNA-
binding protein involved in the regulation of RNA processing. The accumulation of TDP-43 …

[HTML][HTML] Lysosome dysfunction as a cause of neurodegenerative diseases: Lessons from frontotemporal dementia and amyotrophic lateral sclerosis

J Root, P Merino, A Nuckols, M Johnson… - Neurobiology of disease, 2021 - Elsevier
Frontotemporal dementia (FTD) and amyotrophic lateral sclerosis (ALS) are fatal
neurodegenerative disorders that are thought to exist on a clinical and pathological …

Autophagy induction as a therapeutic strategy for neurodegenerative diseases

A Djajadikerta, S Keshri, M Pavel, R Prestil… - Journal of molecular …, 2020 - Elsevier
Autophagy is a major, conserved cellular pathway by which cells deliver cytoplasmic
contents to lysosomes for degradation. Genetic studies have revealed extensive links …

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 …

Functional roles and networks of non-coding RNAs in the pathogenesis of neurodegenerative diseases

YY Wu, HC Kuo - Journal of Biomedical Science, 2020 - Springer
Recent transcriptome analyses have revealed that noncoding RNAs (ncRNAs) are broadly
expressed in mammalian cells and abundant in the CNS, with tissue and cell type-specific …

Astrocyte-derived extracellular vesicles: Neuroreparative properties and role in the pathogenesis of neurodegenerative disorders

R Upadhya, W Zingg, S Shetty, AK Shetty - Journal of controlled release, 2020 - Elsevier
Extracellular vesicles (EVs) released by neural cells play an essential role in brain
homeostasis and the crosstalk between neural cells and the periphery. EVs are diverse …