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 …

Background Short tandem repeat (STR) expansion disorders are an important cause of
human neurological disease. They have an established role in more than 40 different …

Epilepsy is a common neurological disorder, and mutations in genes encoding ion channels
or neurotransmitter receptors are frequent causes of monogenic forms of epilepsy. Here we …

An intronic expansion of GGGGCC repeats within the C9 ORF 72 gene is the most common
genetic cause of amyotrophic lateral sclerosis and frontotemporal dementia (ALS‐FTD) …

Several recent breakthroughs have provided notable insights into the pathogenesis of
amyotrophic lateral sclerosis (ALS), with some even shifting our thinking about this …

People's differences in cognitive functions are partly heritable and are associated with
important life outcomes. Previous genome-wide association (GWA) studies of cognitive …

With the increase in the ageing population, neurodegenerative disease is devastating to
families and poses a huge burden on society. The brain and spinal cord are extraordinarily …

The causes of amyotrophic lateral sclerosis (ALS), a devastating human neurodegenerative
disease, are poorly understood, although the protein TDP-43 has been suggested to have a …

Stress granules (SGs) are dynamic membraneless compartments composed out of RNA-
binding proteins (RBPs) and RNA molecules that assemble temporarily to allow the cell to …

Background Major efforts have been made in the last decade to develop and improve
therapies for proximal spinal muscular atrophy (SMA). The introduction of …