Recent progress of the genetics of amyotrophic lateral sclerosis and challenges of gene therapy

H Wang, LP Guan, M Deng - Frontiers in neuroscience, 2023 - frontiersin.org
Amyotrophic lateral sclerosis (ALS) is a neurodegenerative disorder characterized by the
degeneration of motor neurons in the brain and spinal cord. The causes of ALS are not fully …

Prospects for gene replacement therapies in amyotrophic lateral sclerosis

I Giovannelli, A Higginbottom, J Kirby… - Nature Reviews …, 2023 - nature.com
Amyotrophic lateral sclerosis (ALS) is a devastating and incurable neurodegenerative
disease characterized by the progressive loss of upper and lower motor neurons. ALS …

ALS-associated KIF5A mutation causes locomotor deficits associated with cytoplasmic inclusions, alterations of neuromuscular junctions, and motor neuron loss

L Soustelle, F Aimond, C López-Andrés… - Journal of …, 2023 - Soc Neuroscience
Amyotrophic lateral sclerosis (ALS) is a fatal neurodegenerative disease affecting motor
neurons. Recently, genome-wide association studies identified KIF5A as a new ALS …

An ALS‐associated KIF5A mutant forms oligomers and aggregates and induces neuronal toxicity

J Nakano, K Chiba, S Niwa - Genes to Cells, 2022 - Wiley Online Library
KIF5A is a kinesin superfamily motor protein that transports various cargos in neurons.
Mutations in Kif5a cause familial amyotrophic lateral sclerosis (ALS). These ALS mutations …

Molecular architecture of the autoinhibited kinesin-1 lambda particle

JF Weijman, SKN Yadav, KJ Surridge, JA Cross… - Science …, 2022 - science.org
Despite continuing progress in kinesin enzyme mechanochemistry and emerging
understanding of the cargo recognition machinery, it is not known how these functions are …

Condensate functionalization with microtubule motors directs their nucleation in space and allows manipulating RNA localization

A Cochard, A Safieddine, P Combe… - The EMBO …, 2023 - embopress.org
The localization of RNAs in cells is critical for many cellular processes. Whereas motor‐
driven transport of ribonucleoprotein (RNP) condensates plays a prominent role in RNA …

Kinesin-14 HSET and KlpA are non-processive microtubule motors with load-dependent power strokes

X Liu, L Rao, W Qiu, F Berger, A Gennerich - Nature communications, 2024 - nature.com
Accurate chromosome segregation during cell division relies on coordinated actions of
microtubule (MT)-based motor proteins in the mitotic spindle. Kinesin-14 motors play vital …

Autoinhibition and activation of kinesin-1 and their involvement in amyotrophic lateral sclerosis

K Chiba, S Niwa - Current Opinion in Cell Biology, 2024 - Elsevier
Kinesin-1, composed of kinesin heavy chain and kinesin light chain, is a founding member
of kinesin superfamily and transports various neuronal cargos. Kinesin-1 is one of the most …

Muscleblind-like proteins use modular domains to localize RNAs by riding kinesins and docking to membranes

RP Hildebrandt, KR Moss, A Janusz-Kaminska… - Nature …, 2023 - nature.com
RNA binding proteins (RBPs) act as critical facilitators of spatially regulated gene
expression. Muscleblind-like (MBNL) proteins, implicated in myotonic dystrophy and cancer …

In silico exploration of metabolically active peptides as potential therapeutic agents against amyotrophic lateral sclerosis

TH Fatoki, S Chukwuejim, CC Udenigwe… - International Journal of …, 2023 - mdpi.com
Amyotrophic lateral sclerosis (ALS) is regarded as a fatal neurodegenerative disease that is
featured by progressive damage of the upper and lower motor neurons. To date, over 45 …