CPC C12N 15/113 (2013.01) [A61K 31/122 (2013.01); A61K 31/4745 (2013.01); A61K 31/7105 (2013.01); A61P 25/00 (2018.01); C12N 2310/14 (2013.01); C12N 2320/31 (2013.01)] | 12 Claims |
1. A method of modulating expression of a gene containing expanded tri-nucleotide repeats in a cell, comprising:
administering to the cell a composition comprising an oligonucleotide that is complementary to a nucleic acid encoding SPT4 or SUPT4H, wherein the oligonucleotide is a siRNA, a shRNA, an antisense oligonucleotide, or a chemical reagent that is 6-chloropurine riboside; and
administering a compound that enhances R-loop formation, wherein the compound is selected from a topoisomerase inhibitor or an RNase H inhibitor,
wherein the gene contains a segment of DNA with CAG or CTG tri-nucleotide repeats that are prone to R-loop formation.
|