Gene therapy for ALS: A review

DA Amado, BL Davidson - Molecular Therapy, 2021 - cell.com
Amyotrophic lateral sclerosis (ALS) has historically posed unique challenges for gene-
therapy-based approaches, due to a paucity of therapeutic targets as well as the difficulty of …

Motor neuron vulnerability and resistance in amyotrophic lateral sclerosis

J Nijssen, LH Comley, E Hedlund - Acta neuropathologica, 2017 - Springer
In the fatal disease—amyotrophic lateral sclerosis (ALS)—upper (corticospinal) motor
neurons (MNs) and lower somatic MNs, which innervate voluntary muscles, degenerate …

FUS-dependent liquid–liquid phase separation is important for DNA repair initiation

BR Levone, SC Lenzken, M Antonaci, A Maiser… - Journal of Cell …, 2021 - rupress.org
RNA-binding proteins (RBPs) are emerging as important effectors of the cellular DNA
damage response (DDR). The RBP FUS is implicated in RNA metabolism and DNA repair …

Neural stem cell transplantation for neurodegenerative diseases

R De Gioia, F Biella, G Citterio, F Rizzo, E Abati… - International journal of …, 2020 - mdpi.com
Neurodegenerative diseases are disabling and fatal neurological disorders that currently
lack effective treatment. Neural stem cell (NSC) transplantation has been studied as a …

Motor neuron susceptibility in ALS/FTD

AMG Ragagnin, S Shadfar, M Vidal… - Frontiers in …, 2019 - frontiersin.org
Amyotrophic lateral sclerosis (ALS) is a neurodegenerative disease characterized by the
death of both upper and lower motor neurons (MNs) in the brain, brainstem and spinal cord …

Gene therapy in amyotrophic lateral sclerosis

T Fang, G Je, P Pacut, K Keyhanian, J Gao, M Ghasemi - Cells, 2022 - mdpi.com
Since the discovery of Cu/Zn superoxide dismutase (SOD1) gene mutation, in 1993, as the
first genetic abnormality in amyotrophic lateral sclerosis (ALS), over 50 genes have been …

IGF2 in memory, neurodevelopmental disorders, and neurodegenerative diseases

CM Alberini - Trends in neurosciences, 2023 - cell.com
Insulin-like growth factor 2 (IGF2) emerged as a critical mechanism of synaptic plasticity and
learning and memory. Deficits in IGF2 in the brain, serum, or cerebrospinal fluid (CSF) are …

IGF2: Development, Genetic and Epigenetic Abnormalities

C Sélénou, F Brioude, E Giabicani, ML Sobrier… - Cells, 2022 - mdpi.com
In the 30 years since the first report of parental imprinting in insulin-like growth factor 2 (Igf2)
knockout mouse models, we have learnt much about the structure of this protein, its role and …

Locomotor deficits in a mouse model of ALS are paralleled by loss of V1-interneuron connections onto fast motor neurons

I Allodi, R Montañana-Rosell, R Selvan, P Löw… - Nature …, 2021 - nature.com
ALS is characterized by progressive inability to execute movements. Motor neurons
innervating fast-twitch muscle-fibers preferentially degenerate. The reason for this …

Ageing and exercise‐induced motor unit remodelling

EJ Jones, SY Chiou, PJ Atherton… - The Journal of …, 2022 - Wiley Online Library
A motor unit (MU) comprises the neuron cell body, its corresponding axon and each of the
muscle fibres it innervates. Many studies highlight age‐related reductions in the number of …