Compositions for regulating the expression of sodium voltage-gated channel alpha subunit 2 and use thereof
A vector with a sequence-specific DNA targeting module and transactivator effectively targets SCN2A-expressing cells to restore normal neuronal function, addressing SCN2A-related disorders by increasing gene expression and alleviating symptoms.
JP2026518475APending Publication Date: 2026-06-08リーゲル セラピューティクス インコーポレイティド
Patent Information
- Authority / Receiving Office
- JP · JP
- Patent Type
- Applications
- Current Assignee / Owner
- リーゲル セラピューティクス インコーポレイティド
- Filing Date
- 2024-05-21
- Publication Date
- 2026-06-08
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Figure 2026518475000001_ABST
Abstract
This specification provides compositions and methods for increasing the expression of the sodium voltage-gated channel alpha subunit 2 (SCN2A) gene in SCN2A-expressing cells. Furthermore, this specification provides the use of such compositions for treating neurological disorders associated with SCN2A deficiency.
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