Methods and compounds for the treatment of genetic disease
AssigneeDesign Therapeutics, Inc.
InventorsAseem ANSARI, Pratik SHAH
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Abstract
The present disclosure relates to compounds and methods which may be useful for modulating the expression of a target gene comprising a CGG trinucleotide repeat sequence and treating diseases and conditions in which the target gene plays an active role. The present disclosure provides compounds and methods for modulating the expression of fmr1 and fmr2, and provides compounds and methods for treating fragile X syndrome and fragile XE syndrome.
Claims17
-188. (canceled)
A method for modulating transcription of a gene comprising a trinucleotide repeat sequence CGG, the method comprising contacting a cell comprising the gene with an agent having a first terminus, a second terminus, and an oligomeric backbone, wherein:
- (a) the first terminus comprises a DNA-binding moiety capable of noncovalently binding to the trinucleotide repeat sequence CGG;
- (b) the second terminus comprises a protein-binding moiety capable of binding to a regulatory molecule that modulates an expression of the gene comprising the trinucleotide repeat sequence CGG;
- and
- (c) the oligomeric backbone comprises a linker between the first terminus and the second terminus.
Dependencies are inferred from the claim text.