Technology ID
TAB-3578
A Cell Line that Expresses secNluc and GFP to Recapitulate PMP22 Gene Expression for Studying Peripheral Neuropathy
E-Numbers
E-012-2014-0
Lead Inventor
Inglese, James (NCATS)
Co-Inventors
Svaren, John (University)
Jang, Sung-Wook (NCATS)
Applications
Therapeutics
Research Materials
Therapeutic Areas
Neurology
Lead IC
NCATS
ICs
NCATS
This technology includes a cell line that expresses two reporters (a secreted luciferase, secNLuc, and GFP) in a pattern that recapitulates the endogenous expression of the peripheral myelin protein 22 (Pmp22) gene. Pmp22 is mainly expressed in the Schwann cells of the peripheral nervous system. Many neurological disorders are associated with aberrations in Schwann cells, including the most common inherited peripheral neuropathy known as Charcot-Marie-Tooth (CMT) disease. This cell line will permit the study of the regulatory elements behind the gene.
Commercial Applications
This cell line serves as an indispensable tool to investigate the regulatory circuitry of the Pmp22 gene whose aberrant expression is clinically linked with the most common inherited peripheral neuropathy known as Charcot-Marie-Tooth (CMT) disease. In particular, the utility of the cell line as a cell-based assay platform could significantly enable a large-scale screening campaign to develop treatments for CMT.
Competitive Advantages
Whereas the previous use of reporter genes has little control over the location of the reporter genes in the genome, this cell line has been genetically modified to simultaneously express the reporters secNluc and GFP to recapitulate expression of the Pmp22 gene from its native genomic locus and therefore enhance the physiological representation of the reporter genes.
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