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, thereby downregulating their production.
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 uses miRNA as an intermediary molecule to regulate target gene expression. The recombinant plasmid introduces exogenous miRNA sequences that specifically bind to target mRNA molecules, preventing their translation into bioactive proteins. This intermediary mechanism provides more effective and specific regulation compared to existing therapies, directly addressing the inadequacy in regulation effectiveness while maintaining targeted adaptability through sequence-specific design
2Reliability
If miRNA production is upregulated to decrease target biomolecule bioavailability, then disease treatment is achieved, but complex molecular mechanisms are involved
Solution Approach 1:
The patent segments the therapeutic approach into distinct functional components: the recombinant plasmid vector, the promoter region for transcriptional control, the miRNA coding sequences for specific target recognition, and the RNA processing machinery. This segmentation allows each component to be optimized independently while working together to achieve reliable disease treatment, reducing the perceived complexity through modular design
Solution Approach 2:
The introduced miRNA sequences utilize the cell's endogenous RNA processing and translation machinery to achieve therapeutic effects. The plasmid design includes promoter regions that leverage cellular transcription factors and processing signals that utilize cellular RNA processing enzymes. This self-service approach allows the therapeutic mechanism to operate through the body's existing molecular infrastructure, reducing the complexity burden of introducing entirely new molecular systems
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 upregulate miRNA production within cells, leading to decreased translation and bioavailability of target biomolecules, thus ameliorating dysregulation and preventing or treating associated diseases.
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
a backbone sequence of nucleic acids that facilitates introduction of the one or more insert sequences into one or more of a subject's cells where it is expressed and/or replicated
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.