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Profiles

New York Stem Cell Foundation

The mission of The New York Stem Cell Foundation (NYSCF) is to accelerate cures for the major diseases of our time through stem cell research. Working across many diseases and disciplines, the NYSCF Research Institute enables scientists to take on the major diseases of our time through stem cell research, leading technological and biological innovations that accelerate therapies.

In December 2014, CMTA announced a collaboration with the NYSCF Research Institute to make human stem cell lines that represent the genetic disease defect for a collection of CMT subtypes. These cell lines were derived from patient materials curated through CMTA clinical Centers of Excellence and are now distributed by NYSCF. The immediate use of these cell lines is to develop methods that allow cells to mimic the Type 1 (Schwann cell) or Type 2 (motor nerve) defects seen in CMT patients. The cell lines are being used in therapy evaluation testing by CMTA Alliance Partners, allowing rapid therapeutic candidate testing in patient-centric disease models at a very early stage. Over 37 partners have so far made use of this world-leading CMT biobank.