Artificial Nucleic Acid Molecules With Modified UTR Elements
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Solution Overview
Problem
Current nucleic acid molecules used in gene therapy and genetic vaccination face challenges such as instability, limited expression levels, and risks of undesired genomic integration and antibody generation, particularly when using RNA or DNA.
Innovation Solution
Development of artificial nucleic acid molecules characterized by high translation efficiency, incorporating specific 5'-UTR and 3'-UTR elements that stabilize the mRNA and enhance protein production without compromising stability or immune response.
Engineering Contradictions & Design Principles
Engineering Contradiction Analysis
1Productivity
If RNA or DNA is used in gene therapy and genetic vaccination, then translation efficiency and expression levels can be improved, but stability decreases and risks of undesired genomic integration and antibody generation increase
Solution Approach 1:
The patent modifies the chemical structure of nucleic acid molecules by introducing modified bases and modified phosphodiester bonds. These parameter changes at the molecular level enhance stability against degradation while preserving translation efficiency. The modified phosphodiester bonds specifically protect against nuclease degradation, and the modified bases reduce immunogenicity, thereby resolving the contradiction between improved productivity and worsened reliability.
Solution Approach 2:
The patent creates composite nucleic acid structures by combining modified bases, modified phosphodiester bonds, and specific UTR elements (5'-UTR and 3'-UTR) in a single molecule. This composite approach integrates multiple functional components that collectively enhance stability, maintain translation efficiency, and reduce immunogenicity. The synergistic effect of these composite elements resolves the technical contradiction by providing both improved productivity and reliability simultaneously.
2Productivity
If nucleic acid molecules are used for gene therapy, then expression levels can be enhanced, but immune response issues arise
Solution Approach 1:
The patent modifies the chemical parameters of nucleic acid molecules by introducing modified bases and modified phosphodiester bonds. These changes alter the molecular properties to reduce recognition by the immune system while maintaining high expression levels. The modified chemical structure reduces activation of immune sensors, thereby resolving the contradiction between enhanced productivity and harmful immune responses.
Solution Approach 2:
The patent converts the potential harm of immunogenicity into a benefit by using modified nucleic acid structures that specifically reduce immune recognition. The modified bases and phosphodiester bonds are designed to evade immune detection while maintaining functional activity, effectively converting the harmful immune response into a beneficial reduction of immunogenicity without sacrificing expression levels.
3Device complexity
If conventional nucleic acid molecules are used, then simplicity is maintained, but translation efficiency and stability are limited
Solution Approach 1:
The patent applies parameter changes to the molecular structure by introducing modified bases and modified phosphodiester bonds. These changes enhance translation efficiency and stability without fundamentally altering the overall molecular architecture. The modifications are integrated into the existing nucleic acid framework, maintaining relative simplicity while achieving improved productivity through targeted chemical parameter optimization.
Data Source
AI summary
The invention relates to an artificial nucleic acid molecule comprising at least one open reading frame and at least one 3-untranslated region element (3′-UTR element) and/or at least one 5-untranslated region element (5′-UTR element), wherein said artificial nucleic acid molecule is characterized by high translation efficiency. The translation efficiency is contributed, at least in part, by the 5′-UTR element or the 3′-UTR element, or both of the 5′-UTR element and the 3′-UTR element. The invention further relates to the use of such an artificial nucleic acid molecule in gene therapy and/or genetic vaccination. Furthermore, novel 3′-UTR elements and 5′-UTR elements are provided.