Nucleic acid molecule and use thereof
By designing nucleic acid molecules containing PAH sequences and regulatory polynucleotides, and controlling expression using liver-specific promoters, the compliance and cost issues of existing PKU treatments have been addressed, achieving more effective control of phenylalanine levels and prevention of neurological symptoms.
Patent Information
- Authority / Receiving Office
- WO · WO
- Patent Type
- Applications
- Current Assignee / Owner
- SUZHOU HEGUANG KEHUI BIOTECHNOLOGY CO LTD
- Filing Date
- 2024-12-31
- Publication Date
- 2026-07-09
AI Technical Summary
Current PKU treatments mainly rely on diet management and gene replacement therapy, which have problems such as poor treatment adherence, immune response, long-term medication requirements and high costs. Furthermore, they cannot completely prevent neurological symptoms, and patients need more effective non-dietary treatment options.
A nucleic acid molecule was designed containing a sequence encoding phenylalanine hydroxylase (PAH) and a regulatory polynucleotide, which reduces or inhibits endogenous PAH expression under the control of a liver-specific promoter, thereby improving drug efficacy, and is supplemented by a sequence encoding a normal PAH protein.
It improved drug efficacy, reduced the dosage of transitional drugs, effectively lowered blood phenylalanine levels, reduced reliance on dietary management, and prevented neurological symptoms.
Smart Images
Figure PCTCN2024144389-FTAPPB-I100001 
Figure PCTCN2024144389-FTAPPB-I100002 
Figure PCTCN2024144389-FTAPPB-I100003