News Related to: ALS Association and Target ALS to Expand Tissue Core for ALS Research
A Stem Cell Model of the Motor Circuit Uncouples Motor Neuron Death from Hyperexcitability Induced by SMN Deficiency
In spinal muscular atrophy, a neurodegenerative disease caused by ubiquitous deficiency in the survival motor neuron (SMN) protein, sensory-motor synaptic dysfunction and...
ALS-associated mutant FUS induces selective motor neuron degeneration through toxic gain of function
Mutations in FUS cause amyotrophic lateral sclerosis (ALS), including some of the most aggressive, juvenile-onset forms of the disease. FUS loss-of-function and toxic gain-of...
Role of Primary Afferents in the Developmental Regulation of Motor Axon Synapse Numbers on Renshaw Cells
Motor function in mammalian species depends on the
maturation of spinal circuits formed by a large variety
of interneurons that regulate motoneuron firing and
ALS/FTD Mutation-Induced Phase Transition of FUS Liquid Droplets and Reversible Hydrogels into Irreversible Hydrogels Impairs RNP Granule Function
The mechanisms by which mutations in FUS and other RNA binding proteins cause ALS and FTD remain controversial. We propose a model in which low-complexity (LC) domains of FUS drive its...
Findings point to potential therapeutic targets
NEW YORK, NY (February 19, 2015)
Study in Human Cells May Improve Ability to Identify Useful Drug Targets
NEW YORK, NY (February 6, 2014)
The discovery of key mediators of neurodegenerative phenotypes, especially prior to neurons becoming...
The first symptomatic SMA patient, ever to receive an intrathecal injection of the ISIS Pharmaceuticals SMNRx, was dosed on December 12, 2011 at the Columbia University Medical Center as part of...