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 encoding miRNA sequences that target and degrade 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 introduces miRNA molecules as intermediary agents that specifically bind to target mRNA sequences of complement and factor proteins. These miRNA molecules act as mediators between the therapeutic composition and the target biomolecules, enabling precise post-transcriptional silencing. The miRNA-loaded liposomes serve as delivery intermediaries that transport the miRNA to target cells, resolving the contradiction by providing both reliable inhibition and specific adaptability through sequence-complementary binding.
Solution Approach 2:
The patent changes the regulatory parameter from transcriptional control to post-transcriptional control by introducing miRNA molecules. This parameter change allows for fine-tuned regulation of bioactive molecule expression levels after the mRNA has been transcribed. By altering the stage of gene expression regulation, the therapy achieves both reliability in reducing protein levels and adaptability to target specific mRNA sequences with high precision.
2Quantity of substance
If miRNA production is increased to downregulate target biomolecule expression, then bioavailability of target biomolecule decreases, but this requires effective delivery mechanisms
Solution Approach 1:
Liposomes are used as intermediary delivery vehicles that encapsulate and protect miRNA molecules from degradation while facilitating their uptake by target cells. The liposomal carrier acts as a mediator between the exogenous miRNA and the cellular machinery, enabling efficient delivery without requiring complex viral vectors or other sophisticated delivery systems. This resolves the contradiction by providing simple yet effective delivery that achieves substantial downregulation of target biomolecules.
Solution Approach 2:
The patent utilizes the cell's own endogenous miRNA processing machinery by introducing exogenous miRNA sequences that mimic natural miRNA structures. Once delivered, these synthetic miRNA molecules are processed and functionally integrated into the cell's RNA interference pathway, copying the natural regulatory mechanism. This approach achieves effective delivery and function without requiring complex external delivery systems, as the cell's own machinery handles the rest of the process.
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 by inducing miRNA production, thereby ameliorating dysregulation and preventing or treating associated diseases.
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.