miRNA Expression Composition for Complement and Factor Suppression
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Solution Overview
Problem
Dysregulated expression of bioactive molecules such as complements and factors leads to homeostatic imbalance, resulting in disease, necessitating therapies to regulate their production.
Innovation Solution
Compositions comprising recombinant plasmids encoding miRNA sequences that target and degrade or inactivate the mRNA of specific biomolecules like complements and factors, administered via vectors like AAV, to upregulate miRNA production in cells, thereby reducing the bioavailability of these molecules.
Engineering Contradictions & Design Principles
Engineering Contradiction Analysis
1Quantity of substance
If bioactive molecules are over-expressed, then the quantity of substance increases, but homeostasis is lost and disease occurs
Solution Approach 1:
The patent employs miRNA molecules that provide negative feedback regulation by binding to target mRNA sequences of over-expressed bioactive molecules, thereby reducing their translation and restoring homeostatic balance. The miRNA acts as a feedback mechanism that senses and corrects abnormal protein expression levels.
Solution Approach 2:
The invention changes the expression parameter of target bioactive molecules by introducing miRNA sequences that specifically bind to and suppress the translation of over-expressed proteins, thereby adjusting the quantity parameter back to physiological levels and restoring homeostasis.
2Quantity of substance
If miRNA is used to degrade target mRNA, then the bioavailability of target biomolecule decreases, but the complexity of the composition increases
Solution Approach 1:
The patent uses miRNA as an intermediary molecule that mediates the degradation of target mRNA. Instead of directly eliminating the target protein, the miRNA acts as a mediator that binds to complementary sequences in target mRNA, recruiting cellular machinery to degrade the mRNA and thereby reducing protein production through a natural cellular pathway.
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 and activity within subjects.
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 target mRNA to be degraded or inactivated
Implementation Method 2
administered via vectors like AAV, to upregulate miRNA production in cells
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.