Nucleic acid molecules and uses thereof
TW202635885APending Publication Date: 2026-09-01GRITELEMENT THERAPEUTICS CO LTD
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Patent Information
- Application Number
- TW114152001
- Authority / Receiving Office
- TW · TW
- Patent Type
- Applications
- Current Assignee / Owner
- Priority Date
- 2024-12-31
- Filing Date
- 2025-12-30
- Publication Date
- 2026-09-01
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Figure TWG2TA001075079_001 
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Abstract
This disclosure relates to a nucleic acid molecule comprising (a) a sequence encoding phenylalanine hydroxylase (PAH), and / or (b) a small nucleic acid that reduces or inhibits the expression of endogenous PAH. The disclosure also relates to a vector, a cell, and a pharmaceutical composition comprising the nucleic acid molecule, as well as methods for using them to treat a disease or disorder associated with insufficient phenylalanine hydroxylase activity.
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