miRNA Plasmid Composition for Complement and Factor Downregulation
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Solution Overview
Problem
Dysregulation of bioactive molecules such as complements and factors leads to homeostatic imbalance, resulting in disease, and existing therapies are inadequate for effectively regulating their expression.
Innovation Solution
Compositions comprising recombinant plasmids that encode for micro-interfering ribonucleic acid (miRNA) sequences, which target and degrade or inactivate the mRNA of specific biomolecules like complements and factors, increasing endogenous production of miRNA to downregulate their expression.
Engineering Contradictions & Design Principles
Engineering Contradiction Analysis
1Reliability
If existing therapies are used to regulate bioactive molecule expression, then treatment is provided, but the regulation is inadequate and homeostatic imbalance persists
Solution Approach 1:
The patent uses miRNA as an intermediary molecule to regulate target biomolecule expression. The composition includes miRNA that specifically binds to mRNA of target biomolecules (complements and factors), preventing their translation and achieving precise downregulation of specific proteins while maintaining homeostasis of other bioactive molecules.
Solution Approach 2:
The patent changes the concentration parameter of specific biomolecules by introducing miRNA sequences that target and degrade specific mRNA. This allows selective adjustment of individual biomolecule levels (such as C1q, C3, Factor B) without affecting the overall system, enabling precise homeostatic regulation.
2Quantity of substance
If miRNA production is increased to downregulate target biomolecule expression, then bioavailability of target biomolecule decreases, but this requires effective delivery and expression systems
Solution Approach 1:
The patent employs plasmid DNA that can autonomously replicate and express miRNA sequences once introduced into cells. The system serves itself by utilizing the cell's own transcriptional and translational machinery to produce functional miRNA, eliminating the need for complex continuous delivery systems.
Solution Approach 2:
The patent extracts and isolates specific miRNA sequences that target particular biomolecules, separating the regulation function from the delivery vehicle. The plasmid contains only the essential elements (promoter, miRNA sequence, polyadenylation signal) needed for functional miRNA production, simplifying the overall system design.
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 the bioavailability of target biomolecules, restoring homeostasis and potentially treating associated diseases by enhancing miRNA production within cells.
Implementation Method 1
the miRNA can cause the target mRNA to be degraded or inactivated, thereby causing a decrease in bioavailability of the target biomolecule because it is degraded or inactivated by the miRNA
Data Source
AI summary
Some embodiments of the present disclosure relate to one or more compositions that upregulate the production of one or more sequences of micro-interfering ribonucleic acid (miRNA). The miRNA may be complimentary to a sequence of target messenger RNA (mRNA) that encodes for a target biomolecule and the miRNA can cause the target mRNA to be degraded or inactivated, decreasing the bioavailability of the target biomolecule within a subject that is administered the one or more compositions. In some embodiments of the present disclosure, the target biomolecule is a complement or a factor. In some embodiments of the present disclosure, the target biomolecule is a complement such as complement C1q, complement C1r, complement C1s, complement C3 or complement C5. In some embodiments of the present disclosure, the target biomolecule is a factor such as Factor B, Factor D or Factor 10.