Compounds and methods for modulating frataxin expression
AssigneeWisconsin Alumni Research Foundation
InventorsAseem Ansari, Graham Erwin, Matthew Grieshop
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Abstract
The present technology relates to compositions and methods for modulating expression of genes, which include a target oligonucleotide sequence, such as repeats of a particular oligonucleotide sequence containing 3 to 10 nucleotides. In particular aspects, the present technology relates to agents having a formula A-L-B, wherein -L- is a linker; A- is a Brd4 binding moiety; and -B is a nucleic acid binding moiety, such as a polyamide or complementary oligonucleotide, that specifically binds to the target oligonucleotide sequence.
Claims31
An agent having a formula A-L-B wherein -L- is a linker;
- A- is a bromodomain-containing protein 4 (Brd4) binding moiety having a structure of a bromodomain inhibitor;
- and -B is a nucleic acid binding moiety that specifically binds to one or more repeats of a GAA oligonucleotide sequence.
Dependencies are inferred from the claim text.