Recombinant miRNA Plasmids for Complement mRNA Silencing
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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 are administered to upregulate miRNA production, targeting and degrading or inactivating the mRNA of specific biomolecules like complements and factors, thereby reducing their bioavailability.
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 effectiveness is inadequate
Solution Approach 1:
The patent introduces miRNA as an intermediary molecule that mediates the regulation of bioactive molecules. The recombinant plasmid encodes miRNA sequences that specifically target and bind to mRNA of complement factors (C1q, C1r, C1s, C3, C5, Factor B, Factor D, Factor 10), forming an RNA-RNA hybrid that leads to mRNA degradation or translational inhibition, thereby achieving precise and effective downregulation of the target proteins.
Solution Approach 2:
The patent changes the molecular parameter of gene expression by introducing exogenous miRNA sequences that alter the expression levels of target biomolecules. The recombinant plasmid system enables controlled transcription and processing of miRNA, which then modifies the abundance of specific mRNA transcripts through sequence-specific binding, achieving dynamic regulation of protein expression levels.
2Quantity of substance
If miRNA production is upregulated to degrade target mRNA, then bioavailability of target biomolecule decreases, but this requires effective delivery and expression systems
Solution Approach 1:
The patent employs preliminary action by designing and constructing recombinant plasmids that already contain the necessary regulatory elements (promoters, polyadenylation signals, multiple cloning sites) and miRNA coding sequences before administration. The plasmid is pre-assembled with all required components to ensure efficient nuclear entry, transcription, and miRNA processing upon cellular uptake, eliminating the need for complex in vivo assembly steps.
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 downregulate the expression of target biomolecules, restoring homeostasis and potentially treating associated diseases by increasing 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.