Composition and method for organ-protective expression and regulation of coding ribonucleic acid

By employing mRNA sequences with miRNA binding sites and targeted delivery particles, the method addresses the challenge of differential expression and targeting specificity in delivering polynucleotides to specific organs and tissues, improving treatment efficacy and reducing side effects.

KR103006109B1Active Publication Date: 2026-08-14COMBINED THERAPEUTICS INC
1 Cites 0 Cited by

Patent Information

Application Number
KR1020207024476
Authority / Receiving Office
KR · KR
Patent Type
Patents
Current Assignee / Owner
Priority Date
2018-09-06
Filing Date
2019-02-19
Publication Date
2026-08-14
Estimated Expiration
2039-02-19

AI Technical Summary

Technical Problem

Existing methods for delivering polynucleotide sequences, such as mRNA, to specific organs and tissues face challenges in achieving effective and differential expression in different cell types within the body, with limitations in transfection rates and targeting specificity, and risks of toxicity and inflammation.

Method used

The use of an mRNA sequence with miRNA binding sites adjacent to the UTR sequence, combined with delivery particles targeting specific organs, enables differential expression of polypeptides in different cell types and enhances targeting specificity.

Benefits of technology

This approach allows for controlled and specific expression of therapeutic agents in target organs, reducing side effects and improving treatment efficacy by enhancing transfection rates and organ specificity.

✦ Generated by Eureka AI based on patent content.
Patent Text Reader

Abstract

An isolated mRNA sequence for the expression of one or more polypeptides in one or more target organs, comprising at least one coding sequence encoding at least one polypeptide, at least one first untranslated region (UTR) sequence and a plurality of microRNA (miRNA) binding site sequences. Each miRNA binding site sequence is located immediately adjacent to the 5' or 3' of the first UTR sequence; the miRNA binding site sequence enables differential expression of the coding sequence in at least first and second cell types within the target organ or organs. A method of using the above composition is provided, in particular, for the treatment of diseases such as liver cancer, brain cancer, lung cancer, breast cancer, pancreatic cancer, colon cancer, and kidney cancer.
Need to check novelty before this filing date? Find Prior Art