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Little is known concerning the molecular pathology underlying these conditions, partly because it has been impossible to access significant quantities of the disease-affected cell, the spinal motor neuron.
With recent advances in stem cell and reprogramming biology, we can now produce billions of spinal motor neurons with control and diseased genotypes.
Project ALS has succeeded in assembling one of the most interactive groups of scientists that I’ve ever encountered outside of my department at Harvard.
These relationships, fostered and supported by Project ALS keep the work moving at a speed we could never achieve on our own.
We use this new resource to design in vitro disease models for both mechanistic studies and for the discovery of novel small-molecule therapeutics. Snap Shot: directed differentiation of pluripotent stem cells. Pub Med Mekhoubad S, Bock C, de Boer AS, Kiskinis E, Meissner A, Eggan K.
A number of devastating diseases, including amyotrophic lateral sclerosis (ALS) and spinal muscular atrophy (SMA), specifically affect the neuromuscular system.
Reference Maps of human ES and i PS cell variation enable high-throughput characterization of pluripotent cell lines. Pub Med Son EY, Ichida JK, Wainger BJ, Toma JS, Rafuse VF, Woolf CJ, Eggan K.
Eggan Lab Homepage Members of the Eggan Lab List of Publications from Pub Med A fundamental understanding of how a cell's identity is determined during differentiation and how it can in turn be manipulated experimentally is a central goal of developmental biology, one with substantial ramifications for biomedicine.
We study both the differentiation of embryonic stem cells into the neural lineage and the reprogramming of commonly available differentiated cell types, such as fibroblasts, into either pluripotent stem cells or cells of therapeutic interest, such as spinal motor neurons.
The idea that we might use embryonic stem cells to produce the motor neurons that die in ALS was an exciting idea, but no one believed that we would be able to do it. In contrast, Project ALS had the foresight to realize what a powerful approach this was.
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