Recombinant miRNA Plasmid Composition for Target mRNA Suppression
Find Innovative SolutionsGenerate Solutions
Solution Overview
Problem
Bioactive molecule overexpression or mis-expression leads to homeostasis disruption and disease, necessitating therapies to regulate gene expression and suppress bioactive molecule production.
Innovation Solution
Compositions comprising recombinant plasmids encoding miRNA sequences that target and degrade or inactivate mRNA of specific biomolecules, administered via vectors like AAV, to upregulate miRNA production and decrease target biomolecule synthesis.
Engineering Contradictions & Design Principles
Engineering Contradiction Analysis
1Quantity of substance
If bioactive molecules are over-expressed or mis-expressed, then the production of interfering RNA increases, but homeostasis is lost and disease occurs
Solution Approach 1:
The patent converts the harmful effect of over-expressed bioactive molecules into a benefit by utilizing the cell's natural RNA interference mechanism. Synthetic double-stranded RNA molecules are designed to match the sequence of the over-expressed target mRNA, triggering the cell's endogenous RNAi pathway to degrade the target mRNA and reduce protein production, thereby restoring homeostasis while leveraging the natural interference RNA production mechanism.
Solution Approach 2:
The patent introduces synthetic double-stranded RNA molecules as intermediaries that mediate between the therapeutic goal (reducing over-expressed protein) and the cellular mechanism (RNA interference). These synthetic RNA molecules serve as the intermediary that enters the cell, binds to target mRNA, and activates the endogenous RNAi machinery to achieve the desired therapeutic effect.
2Quantity of substance
If miRNA production is upregulated to degrade target mRNA, then bioavailability of target biomolecule decreases, but the mechanism requires specific sequence complementarity
Solution Approach 1:
The patent applies parameter changes by modifying the sequence parameters of the synthetic RNA molecules to ensure precise complementarity with the target mRNA. The design process involves selecting and optimizing specific nucleotide sequences that maximize binding affinity and specificity to the target mRNA while minimizing off-target effects, thereby achieving effective degradation with high sequence precision.
3Quantity of substance
If recombinant plasmids are administered to increase miRNA production, then gene expression is regulated, but the delivery system requires vector integration
Solution Approach 1:
The patent extracts the essential functional element (the RNA interference mechanism) from the complex vector delivery system. Instead of relying on permanent genetic integration, the approach uses transient delivery of synthetic double-stranded RNA molecules that can induce RNA interference without requiring complex plasmid integration into the genome, thereby simplifying the delivery system while maintaining therapeutic efficacy.
Applied Scientific Principles
This section explains which scientific principles are used to turn an abstract innovation direction into a practical engineering solution.
Function Achieved in This Case
Regulates gene expression by increasing miRNA production, reducing bioavailability of target biomolecules, thereby ameliorating diseases caused by their overexpression or mis-expression.
Implementation Method 1
The sequences of miRNA may be complimentary to a sequence of target messenger RNA (mRNA) that encodes for translation of a target biomolecule and the miRNA can cause the bioavailability of the target mRNA to be decreased because it is degraded or inactivated by the miRNA
Data Source
AI summary
The embodiments of the present disclosure relate to decreasing the bioavailability of one or more target biomolecules by providing a composition that comprises a recombinant plasmid with one or more sequences of micro interfering ribonucleic acid (miRNA). When the recombinant plasmid interacts with a target cell, it causes the target cell to upregulate production of the miRNA, which then decreases the bioavailability of the target biomolecule. In some embodiments of the present disclosure, the target biomolecule is a protein that participates in one or more intracellular processes.