Motor neuron (MN) diseases, including amyotrophic lateral sclerosis, progressive bulbar
palsy, primary lateral sclerosis and spinal muscular atrophy, cause progressive paralysis …

Within mammalian systems, there exists enormous opportunity to use synthetic gene circuits
to enhance phenotype-based drug discovery, to map the molecular origins of disease, and …

Introduction Intronic repeat expansions in the C9orf72 gene are the most frequent known
single genetic causes of amyotrophic lateral sclerosis (ALS) and frontotemporal dementia …

Amyotrophic Lateral Sclerosis (ALS) is a motor neurodegenerative disorder whose cellular
hallmarks are the progressive death of motor neurons (MNs) located in the anterior horn of …

Motor neurons are large projection neurons classified into upper and lower motor neurons
responsible for controlling the movement of muscles. Degeneration of motor neurons results …

Despite mediating several essential processes in the brain, including during development,
cyclin-dependent kinase-like 5 (CDKL5) remains a poorly characterized human protein …

Abstract Coenzyme Q 10 (CoQ 10) is a critical component of the mitochondrial respiratory
chain. CoQ 10 deficiencies often cause a variety of clinical syndromes, often involving …

Introduction ALS is a fatal neurodegenerative disease for which underlying mechanisms are
incompletely understood. The motor neuron is a central player in ALS pathogenesis but …

Neuromuscular diseases (NMDs) are a genetically or clinically heterogeneous group of
diseases that involve injury or dysfunction of neuromuscular tissue components, including …

Introduction Cell therapy development represents a critical challenge in amyotrophic lateral
sclerosis (ALS) research. Despite more than 20 years of basic and clinical research, no …