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1. (WO2018106782) METHODS AND COMPOSITIONS FOR ENHANCING FUNCTIONAL MYELIN PRODUCTION

Pub. No.:    WO/2018/106782    International Application No.:    PCT/US2017/064870
Publication Date: Fri Jun 15 01:59:59 CEST 2018 International Filing Date: Thu Dec 07 00:59:59 CET 2017
IPC: C12N 15/10
C07K 14/47
C12N 15/63
C12N 15/864
Applicants: CASE WESTERN RESERVE UNIVERSITY
Inventors: TESAR, Paul
ELITT, Matthew
Title: METHODS AND COMPOSITIONS FOR ENHANCING FUNCTIONAL MYELIN PRODUCTION
Abstract:
A method of generating a cell that enhances functional myelin production is provided, the method including genetically modifying the cell such that: (i) an endogenous PLP1 gene is modified to decrease its ability to inhibit myelin production; (ii) an endogenous PLP1 genetic regulatory element is modified to decrease its ability to promote PLP1 expression; (iii) an endogenous PLP1 genetic regulatory element is modified to increase its ability to inhibit PLP1 expression; or (iv) an endogenous PLP1 gene product or a PLP1 regulatory element gene product that promotes PLP1 expression is modified to decrease thePLP1 expression level, wherein the cell produces functional myelin.