Recombinant miRNA Plasmid Composition for Target Biomolecule Suppression
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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 that encode miRNA sequences, administered via vectors like AAV, to upregulate miRNA production, targeting and degrading or inactivating target mRNA, thereby reducing bioactive molecule levels.
Engineering Contradictions & Design Principles
Engineering Contradiction Analysis
1Reliability
If bioactive molecules are over-expressed or mis-expressed, then disease prevention or treatment is achieved, but homeostasis is disrupted
Solution Approach 1:
The patent employs feedback control through engineered miRNA molecules that detect and respond to abnormal protein levels. The miRNA expression is regulated to suppress bioactive molecules only when over-expressed, creating a feedback loop that restores homeostasis rather than disrupting it. This is achieved through inducible promoters and titratable miRNA expression systems that adjust suppression levels based on cellular conditions.
Solution Approach 2:
The patent changes the expression parameters of bioactive molecules by introducing miRNA that targets specific mRNA sequences. By adjusting miRNA concentration, sequence complementarity, and expression timing, the system precisely controls protein levels to prevent disease while maintaining homeostasis. The parameter changes are reversible and tunable, allowing dynamic adjustment between health and disease states.
2Quantity of substance
If miRNA production is upregulated to suppress target biomolecule, then bioactive molecule levels decrease, but off-target effects may occur
Solution Approach 1:
The patent applies local quality by designing miRNA molecules with highly specific seed sequences that match only the intended target mRNA. The miRNA expression is localized to specific cell types through tissue-specific promoters, ensuring that suppression occurs only in the relevant tissue context. This spatial and sequence-specificity minimizes off-target effects while maintaining effective target suppression.
Solution Approach 2:
The patent uses partial action by employing miRNA molecules with imperfect complementarity to target mRNA, which allows for controlled suppression without complete degradation. This partial binding affinity enables fine-tuned regulation that reduces off-target effects compared to complete matches, while still achieving sufficient suppression of the target biomolecule to prevent disease.
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
The compositions effectively decrease target biomolecule production, restoring homeostasis and potentially treating associated diseases by increasing miRNA production within cells.
Implementation Method 1
compositions comprise a plasmid of deoxyribonucleic acid (DNA) that includes one or more insert sequences of nucleic acids that encode for the production of miRNA
Implementation Method 2
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
Implementation Method 3
administered via vectors like AAV
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.