Optimized gapmers antisense oligonucleotides for increasing foxg1 expression
AssigneeEliGab Tx LLC
InventorsScott REICH, Brian BETTENCOURT
Life of this patent
Estimating the term…
Abstract
Provided herein are compositions and methods for treating and/or ameliorating the FOXG1 syndrome or the symptoms associated therewith. The compositions and methods disclosed herein utilize antisense oligonucleotides that target long non-coding RNAs (lncRNAs) to increase FOXG1 expression in a cell, thereby restoring FOXG1 function.
Claims43
A gapmer antisense oligonucleotide comprising a sequence that hybridizes to a target nucleic acid sequence of a long non-coding RNA (lncRNA), wherein the gapmer antisense oligonucleotide comprises a structure having the formula:
- wherein:
- X is a 5′ wing region,
- Y is a gap region, and
- Z is a 3′ wing region;
- and
- wherein X, Y, and/or Z comprise one or more modifications.
The method of any one of claims 31 to 38, wherein modulating expression comprises increasing translation of a FOXG1 protein in the cell.
Dependencies are inferred from the claim text.