Methods and compositions for enhancing functional myelin production
AssigneeCase Western Reserve University
InventorsPaul Tesar, Matthew Elitt
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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.
Claims22
A method of sufficiently restoring functional myelin production in a cell in a subject, wherein the subject has insufficient myelination, the method comprising delivering to the cell in the subject an RNAi construct, or a polynucleotide encoding said RNAi construct, to decrease expression level of an endogenous PLP1 gene, thereby sufficiently restoring functional myelin production by the cell, or by a differentiated progeny of the cell, wherein the endogenous PLP1 gene is a deleterious-disease causing mutant PLP1 gene.
A method of treating a myelin related disorder in a subject, wherein the subject has insufficient myelination, the method comprising administering to the subject an RNAi construct or a polynucleotide encoding said RNAi construct, to decrease expression level of an endogenous mutant PLP1 gene of a myelin-producing cell in the subject, thereby sufficiently restoring production of functional myelin in the subject, wherein the myelin related disorder is characterized by misfolded PLP1 protein or abnormal myelination caused by the endogenous mutant PLP1 gene.
Dependencies are inferred from the claim text.