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US 2025/0082780 A1Published application
Compositions for modulating expression of sodium voltage-gated channel alpha subunit 1 and uses thereof
AssigneeRegel Therapeutics, Inc.
InventorsJordane DIMIDSCHSTEIN, Navneet MATHARU
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Abstract
Provided herein are compositions and methods for increasing expression of the sodium voltage-gated channel alpha subunit 1 (SCN1A)gene in SCN1A-expressing cells. Further provided herein are uses of such compositions to treat disorders associated with SCN1A expression deficiency, such as Dravet syndrome.
Claims66
A vector comprising:
- (a) a transgene polynucleotide sequence encoding a sequence-specific DNA-targeting module (DTM) fused to a transactivator;
- (b) an enhancer polynucleotide sequence that specifically restricts expression of the transgene to sodium voltage-gated channel alpha subunit 1 (SCN1A)-expressing cells in the brain;
- and
- (c) a promoter polynucleotide sequence.
1 more element
Dependencies are inferred from the claim text.