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Compound protects nerve cells targeted by neurodegenerative diseases

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SMN protein (red) is necessary for the survival of spinal cord neurons (motor neurons) responsible for breathing and all movement. Harvard researchers have found a compound that stabilized this protein in mouse and human motor neurons. This may lead to...

A new high-throughput platform for discovering potential ALS drugs

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HSCI researchers use patient-derived motor neurons to screen for clinically relevant targets Harvard Stem Cell Institute (HSCI) researchers led by Clifford Woolf, M.D., Ph.D., have developed a high-throughput platform for discovering drug targets to treat...

Gut microbiome influences ALS outcomes

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HSCI scientists identify gut-brain connection in ALS By Jessica Lau HSCI scientists have identified a new gut-brain connection in the neurodegenerative disease amyotrophic lateral sclerosis, or ALS. The researchers found that in mice with a common ALS...

Patient stem cells help identify common problem in ALS

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Discovery will lead directly to clinical trials Harvard stem cell scientists have discovered that a recently approved medication for epilepsy may possibly be a meaningful treatment for amyotrophic lateral sclerosis (ALS)—Lou Gehrig’s disease, a uniformly...

Harvard Stem Cell Institute and Evotec collaborate to advance ALS research

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The Harvard Stem Cell Institute (HSCI) has entered into a strategic partnership with Evotec AG to identify compounds that prevent or slow down the loss of motor neurons, which is characteristic of amyotrophic lateral sclerosis (ALS), also known as Lou...

New use for stem cells identifies a promising way to target ALS

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Using a new, stem cell-based, drug-screening technology that could reinvent and greatly reduce the cost of developing pharmaceuticals, researchers at the Harvard Stem Cell Institute (HSCI) have found a compound that is more effective in protecting the...

HSCI researchers turn one form of neuron into another in the brain

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A new finding by Harvard stem cell biologists turns one of the basics of neurobiology on its head by demonstrating that it is possible to turn one type of already differentiated neuron into another within the brain. The discovery by Paola Arlotta and...

From skin cells to motor neurons: researchers find success with direct cellular reprogramming

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A team of Harvard stem cell researchers has succeeded in reprogramming adult mouse skin cells directly into the type of motor neurons damaged in amyotrophic lateral sclerosis (ALS), best known as Lou Gehrig’s disease, and spinal muscular atrophy (SMA)...

Accelerating a treatment for ALS

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Clinical trial success based on stem cell models in the lab Summary A clinical trial, supported in part by the Harvard Stem Cell Institute (HSCI), has shown that the drug retigabine improves a characteristic of motor neurons that is impacted in...

Potential therapeutic target for ALS

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Discovery opens doors to improving diagnostics and developing new therapy for majority of ALS patients Summary HSCI-funded research using human pluripotent stem cell models and tissue from ALS patients found that the protein TDP-43 regulates a gene called...

HSCI researchers identify another potential ALS treatment

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Similar results in experimental animals and human cells in dish A series of studies begun by Harvard Stem Cell Institute (HSCI) scientists eight years ago has led to a report, published in Science Translational Medicine, that may be a major step forward...

Harvard University President Drew Faust takes ALS ice bucket challenge

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President Drew Faust accepts the ALS Ice Bucket Challenge and is doused by Harvard’s ALS research team, led by Harvard Stem Cell Institute Principal Faculty member Kevin Eggan, PhD. She nominates MIT president Rafael Reif, UMass Boston Chancellor Keith...