Center of Excellence for Myelin Disorders: Mechanisms & Repair
To provide a center for personalized treatment of demyelinating disorders occurring in adults (i.e., in patients who suffer from multiple sclerosis) and children (i.e., Canavan’s disease, genetic, or nutritional disorders).
The emphasis of the center will be on repair.
The strategy includes the collaboration of several scientists with a variety of expertise to identify novel molecular targets that can aid in repairing axons damaged in the absence of myelin and promoting the formation of new myelin.
An additional strength of the center is the generation of stem cells from skin biopsies. The ultimate goal is the implementation of induced pluripotent stem (iPS) cell-based therapies to repair damaged myelin and the study of their proliferative and differentiative potential.
Interdisciplinary Research with a Real Translational Endpoint
Translational research implies the development of new treatments (i.e., target identification, target validation in animal models, and pilot studies in human subjects), as well as the evaluation of current clinical trials.
Based on the current strengths at Mount Sinai (i.e., Corinne Goldsmith Dickinson Center for Multiple Sclerosis and the Genetics Clinical Department), the areas of interest will be repair of lost myelin in white matter disorders in the adult brain (i.e., in patients who suffer from multiple sclerosis) and in children with genetic or metabolic disorders affecting myelin formation.
The idea is to create groups that can synergize efforts and lead to the identification of molecular targets and cell-based strategies to be implemented into phase I clinical trials.
Success is best achieved by identifying specific projects and having clear areas of development in mind. The Center for Excellence for Myelin Disorders: Mechanisms and Repair consists of basic neuroscientists, chemical engineers, and bioinformaticians working together with clinical neurologists at the Corinne Goldsmith Dickinson Center for Multiple Sclerosis research and with clinical geneticists for research on developmental disorders of myelin formation.
Additional areas include the generation and characterization of patient-derived iPS cells, the characterization of stem cell behavior, and neuroprotection in a broad sense.
Patrizia Casaccia, MD, PhD is the Chief for the Center of Excellence on Myelin Disorders: Mechanisms & Repair, Professor of Neuroscience, Genetics and Genomic Sciences and Neurology.
Icahn Medical Institute Floor 10 Room 70F
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New York, NY 10029