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 fatal neurodegenerative disorder. The researchers are now collaborating with Massachusetts ...
|Scooped by Jacob Blumenthal|
In two papers, published in Cell Reports, and in Cell Stem Cell, researchers used induced pluripotent stem cells (iPSC) derived from Amyotrophic Lateral Sclerosis (ALS) patients cells to study aberrant molecular mechanisms related to ALS in-vitro. In the first paper, researchers from the labs of Kevin Eggan and Clifford Woolf found that iPSC-derived motor neurons from ALS patients are hyperexcitable compared to controls, and that the drug Retigabine, which has recently been approved as an anticonvulsive, could rescues the hyperexcitability phenotype in multiple ALS variants.
In the second paper, researchers from the lab of Su-Chun Zhang found that neurofilament aggregation is an early phenotype of ALS, and that correction of neurofilament expression, mitigates axonal degeneration in ALS motor neurons.
Learn about motor neurons development, pathologies, gene expression and related motor neurons derivation protocols: