News
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Research
The Ubiquitination of PINK1 Is Restricted to Its Mature 52-kDa Form
Along with Parkin, PINK1 plays a critical role in maintaining mitochondrial quality control. Although PINK1 is expressed constitutively, its level is kept low in healthy mitochondria by...
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Research Unveils a Developmental Splicing Program Controlling Neuronal Maturation and Excitability
In a new study published in Neuron, Professors Chaolin Zhang and Hynek Wichterle determined that loss of Rbfox genes results in an “embryonic like” splicing program. Importantly, they observed a...
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Verge Genomics Announces a Multi-Institution Collaboration with Columbia University, MGH, USC, and University of Michigan to Advance ALS Research
An innovative industry-academic collaboration combining machine learning with genomic, genetic, biological, and phenotypic data to accelerate the translation of pre-clinical models of ALS
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The C9ORF72 GGGGCC expansion forms RNA G-quadruplex inclusions and sequesters hnRNP H to disrupt splicing in ALS patient brains
An expanded GGGGCC hexanucleotide in C9ORF72 (C9) is the most frequent known cause of
amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD). It has been... -
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...
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Research | Patient Care
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...
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Research
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
motor output... -
Research
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...
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Research | Patient Care
ALS Association and Target ALS to Expand Tissue Core for ALS Research
The ALS Association and Target ALS are pleased to announce the launch of a new collaborative effort to expand the collection of biofluids from people with ALS, to be used for research to better...
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Research
New ALS Gene and Signaling Pathways Identified
Findings point to potential therapeutic targets
NEW YORK, NY (February 19, 2015)
