AI Therapeutics Announces Initiation of a Phase II Clinical Trial of AIT-101 for Treatment of ALS

AI Therapeutics, Inc., a clinical-stage biopharmaceutical company developing novel therapeutics for rare diseases, announced the initiation of a Phase II study for a promising new approach to treat ALS.
[AI Therapeutics, Inc.]
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Astrocytes Display Cell Autonomous and Diverse Early Reactive States in Familial Amyotrophic Lateral Sclerosis

The authors revealed that reactive transformation can occur cell autonomously in human ALS astrocytes and with a striking degree of early molecular and functional heterogeneity when comparing different disease-causing mutations.
[Brain]
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PharmaTher Announces FDA Approval of Investigational New Drug (IND) Application for Ketamine to Treat ALS

PharmaTher Holdings Ltd. announced that the US FDA has accepted an investigator-initiated IND application to proceed with a Phase II clinical trial evaluating ketamine in the treatment of Amyotrophic Lateral Sclerosis (ALS).
[PharmaTher Holdings Ltd.]
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Effects of Intracellular Calcium Accumulation on Proteins Encoded by the Major Genes Underlying Amyotrophic Lateral Sclerosis

Researchers aimed to find a relationship between the proteins coded by the most relevant genes associated with amyotrophic lateral sclerosis and intracellular Ca2+ accumulation.
[Scientific Reports]
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Human Stem Cell Models of Neurodegeneration: From Basic Science of Amyotrophic Lateral Sclerosis to Clinical Translation

The authors evaluate the status of modeling neurodegenerative diseases using iPSCs, including methods for deriving and using disease-relevant neuronal and glial lineages.
[Cell Stem Cell]
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University of Wisconsin Ophthalmology Professor Receives $75,000 Grant to Support International Collaborations on Eye Research

Dr. Steven DeVries, a professor of Ophthalmology at the University of Wisconsin, has received an International Research Collaborators Award from Research to Prevent Blindnes that will allow him to learn from scientists at Ritsumeikan University in Japan about the process of growing 3D retinal organoids from stem cells.
[Ophthalmology Times]
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Novel Chemical Inhibitor against SOD1 Misfolding and Aggregation Protects Neuron-Loss and Ameliorates Disease Symptoms in ALS Mouse Model

Using ELISA-based chemical screening with wild and mutant superoxide dismutase (SOD1) proteins, the authors screened a new small molecule, PRG-A01, which could block the misfolding/aggregation of SOD1 or TDP-43. The drug rescued the cell death induced by mutant SOD1 in human neuroblastoma cell line.
[Communications Biology]
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Axonal TDP-43 Condensates Drive Neuromuscular Junction Disruption through Inhibition of Local Synthesis of Nuclear Encoded Mitochondrial Proteins

The authors showed TDP-43 accumulation in intra-muscular nerves from amyotrophic lateral sclerosis (ALS) patients and in axons of human iPSC-derived motor neurons of ALS patients, as well as in motor neurons and neuromuscular junctions of a TDP-43 mislocalization mouse model.
[Nature Communications]
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A Selectable All-in-One CRISPR Prime Editing piggyBac Transposon Allows for Highly Efficient Gene Editing in Human Cell Lines

Investigators demonstrated proof-of-concept for piggyBac prime-editing in a newly designed lentiviral traffic light reporter, which allowed for estimation of gene correction and defective editing resulting in indels, based on expression of two different fluorophores.
[Scientific Reports]
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Gain-of-Function Cardiomyopathic Mutations in RBM20 Rewire Splicing Regulation and Re-Distribute Ribonucleoprotein Granules within Processing Bodies

To understand the mechanism of RBM20-associated dilated cardiomyopathy (DCM), researchers engineered isogenic iPSCs with DCM-associated missense mutations in RBM20 as well as RBM20 knockout iPSCs.
[Nature Communications]
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Insights of Extracellular Vesicles of Mesenchymal Stem Cells: A Prospective Cell-Free Regenerative Medicine for Neurodegenerative Disorders

Due to paucity of standard protocols in extraction procedures of extracellular vesicles (EVs) and their pharmacological properties and mechanisms, the development of new EV dependent therapies is challenging.
[Molecular Neurobiology]
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