Targeted SECIS RNA mediating UGA codon readthrough and application thereof

By designing targeted SECIS RNA, the secondary structural characteristics of the SECIS element of selenoprotein GPX4 are used to solve the problem of low read-through efficiency of UGA codons in the prior art, effective treatment of nonsense mutation diseases, and further improve read-through efficiency through the combined use of other drugs.

CN119709752APending Publication Date: 2025-03-28CHINA PHARM UNIV
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Patent Information

Application Number
CN202411966611.1
Authority / Receiving Office
CN · China
Patent Type
Applications(China)
Current Assignee / Owner
Filing Date
2024-12-30
Publication Date
2025-03-28

AI Technical Summary

Technical Problem

The prior art has not yet provided an effective and safe method to mediate the read-through of UGA codons and solve the problem of disease treatment caused by nonsense mutations.

Method used

A targeted SECIS RNA was designed to ensure that the secondary structure of the SECIS element remains unchanged by adding Locked sequences and Linker sequences to the SECIS element of selenoprotein GPX4, and to add targeted sequences at its 3' end to improve the efficiency of UGA codon reading.

Benefits of technology

Effectively mediate the read-through of UGA codons, improves the therapeutic potential of nonsense mutation diseases, and further improves the read-through efficiency through combined use with sodium selenite or G418.

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Abstract

The invention belongs to the technical field of genetic engineering and biological medicines, and discloses targeted SECIS RNA (single-electron-chain isotropic system) capable of mediating UGA (uridine-glycolide) codon read-through and application of the targeted SECIS RNA in preparation of read-through treatment medicines or pharmaceutical compositions for nonsense mutation diseases. The targeted SECIS RNA is designed according to SECIS elements of selenoprotein GPX4; the HEK293 cell which is constructed by the invention and can stably express a UGA mutant dual-luciferase reporter gene system is utilized, and dual-luciferase expression detection verifies that the targeting SECIS RNA disclosed by the invention can effectively mediate reading of a UGA codon.
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