Methods and compositions for enhancing functional myelin production
AssigneeCase Western Reserve University
InventorsPaul Tesar, Matthew Elitt
Life of this patent
Estimating the term…
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 the PLP1 expression level, wherein the cell produces functional myelin.
Claims20
A method of generating a cell that enhances functional myelin production, the method comprising delivering to the cell an antisense oligonucleotide (ASO) or a polynucleotide encoding said ASO, to decrease expression level of an endogenous PLP1 gene, wherein the cell produces functional myelin, or is a progenitor that produces or differentiates into the cell that produces functional myelin.
Dependencies are inferred from the claim text.