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NYSCF Collaborates with Charcot-Marie-Tooth Association on Stem Cell Biobank

NEW YORK (GenomeWeb) – The New York Stem Cell Foundation Research Institute today announced a collaboration with the Charcot-Marie-Tooth Association to develop a biobank of stem cell lines to study the group of genetic neuropathies.

NYSCF will use its Global Stem Cell Array technology to create the induced pluripotent stem cell lines, using samples from CMT patients that have been collected at the University of Iowa. The stem cell lines, representing a number of disease states, will then be used to produce differentiated cells that mimic the defective cell types that characterize the different CMT disorders.

As part of the collaboration, NYSCF will make the cell lines available to the global scientific community to enable research and drug development, the partners said.

CMT is a set of rare and disabling neurological disorders of genetic origin. Type 1 is characterized by defective myelin-producing Schwann cells while Type 2 is characterized by defective motor and sensory neurons. In 2010, researchers used whole-genome sequencing to discover the cause of CMT.

The ultimate goal of the collaboration between NYSCF and CMTA is to create a personalized medicine approach to treat the disease.

"Currently, there are no therapies for the different CMT disorders to halt either the onset or progression of the disease," CMTA CEO Patrick Livney said in a statement. "This NYSCF collaboration represents an exciting opportunity for the CMTA to place research on therapies for Charcot-Marie-Tooth disorders in a personalized, patient context at a very early stage."

Financial details of the collaboration were not disclosed.