Translational Advances

The CMTA’s research is done in teams consisting of academic labs and clinical centers sponsored by the CMTA (STAR members), working together and with alliance partners in consortium team efforts. STAR Teams are sponsored by the CMTA via a rapid process of expert review, following invitation of proposals for targeted translational efforts. All sponsorship aims to directly aid the advancement of CMT therapies. The STAR Team approach has in recent years been expanded to include commercial partners expert in creating, breeding and testing animal models of CMT, and an alliance has been formed with the New York Stem Cell Foundation (NYSCF) to generate human stem cell models of CMT disease, starting with cells taken from CMT patients. The cell and animal models, used inside the STAR consortium and made available to alliance partners, now represent a therapy development “toolbox” for several CMT disorders.

STAR Research Translational Advances
	2012	2019
	Cell based reporter assay established for screening of potential therapeutic libraries One potential therapeutic target class identified	Multiple therapy development alliances established Animal models licensed, validated and in use for the evaluation of candidate therapies Expansion of assay capability to include high throughput assays and human patient-derived stem cells made in NYSCF collaboration
	No discussions or assets	Authentic animal models representing human mutation and pathology are available Patient-derived human stem cell lines available through NYSCF
	Planning to test stem cells in animal models	Role of inflammation in disease pathology established Proof of principle in animal model showing inflammatory mediator can regulate disease progression Gene replacement shown to have benefit in an animal model Patient-derived human stem cell lines available through NYSCF
	Struggle to establish animal models No guiding hypothesis for disease intervention	Rodent models established for two human mutations; a strong age-dependent disease pathology is observed Cell line model for phenotypic screening of candidate therapeutics being developed that aims to test role of MFN1 in correcting disease pathology Patient-derived human Stem cell lines available through NYSCF Human stem cell assays being developed to evaluate the cellular pathology of CMT2A
	No animal model available No guiding hypothesis of disease intervention	Authentic rodent model of a human mutation licensed and characterized Drug testing started using stem cells from the animal Patient-derived human stem cell lines available through NYSCF Human stem cell assays being developed to evaluate the cellular pathology of CMT2E
	No discussions	Research investigation of gene therapy for 4C shows therapeutic benefit Patient-derived human stem cell lines available through NYSCF

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