Embryonic Stem Cells from Animal Models of Motor Neuron Diseases Including Spinal Muscular Atrophy (SMA) and Amyotrophic Lateral Sclerosis (ALS)

Progress in the development of therapies for motor neuron diseases has been hampered by the lack of effective cell culture models. Since spinal motor neurons selectively die in ALS and SMA, an effective cell culture model must have mature motor neurons that have the genetic defect found in humans with these disorders and must have an identifiable phenotype that allows them to be distinguished from normal motor neurons in culture. This invention describes generation of embryonic stem (ES) cells from animal models of Amyotrophic Lateral Sclerosis (ALS) and Spinal Muscular Atrophy (SMA) and the differentiation of these cells specifically into motor neurons in culture. The inventors have demonstrated that the resulting motor neurons exhibit many of the pathologic features of the human disease in culture. This technology offers a number of significant advantages over existing methods, including: (a) the ability to generate large numbers of ES cells corresponding to specific motor neuron diseases; (b) the ability to differentiating ES cells into post-mitotic mature motor neurons ; (c) the ability to use fluorescent markers for the rapid identification of such differentiated motor neurons in the culture system; and (d) the ability to characterize abnormal features of motor neurons from animal models of ALS and SMA. Description (Set) Proposed Use (Set) The potential uses of this system are in screening new drugs for motor neuron diseases and in biologic investigation of the molecular causes of these diseases.

Inventor(s): Kerr, Douglas

Type of Offer: Licensing



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