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 …

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 …

Amyotrophic lateral sclerosis (ALS) is a fatal, multigenic, multifactorial, and non-cell
autonomous neurodegenerative disease characterized by upper and lower motor neuron …

Background AG 4 C 2 hexanucleotide repeat expansion in the noncoding region of C9orf72
is the major genetic cause of frontotemporal dementia and amyotrophic lateral sclerosis …

Abstract A G4C2 hexanucleotide repeat expansion in the C9orf72 gene is the most common
genetic cause of ALS and FTLD (C9-ALS/FTLD) with cytoplasmic TDP-43 inclusions …

Synaptic loss is a pathological feature of all neurodegenerative diseases including
amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD). ALS is a disease of …

To better understand human health and disease, researchers create a wide variety of mouse
models that carry human DNA. With recent advances in genome engineering, the targeted …

Genome sequencing of both sporadic and familial patients of Amyotrophic Lateral Sclerosis
(ALS) has led to the identification of new genes that are both contributing and causative in …

Amyotrophic lateral sclerosis (ALS) is a fatal neurological disease, which is characterized by
the degeneration of motor neurons in the motor cortex and the spinal cord and subsequently …

The most common genetic cause of amyotrophic lateral sclerosis (ALS) and frontotemporal
dementia (FTD) is a hexanucleotide expansion in the chromosome 9 open reading frame 72 …