miRNA Plasmid Composition for CCR5 Expression Suppression
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Solution Overview
Problem
Dysregulation of bioactive molecules, such as chemokine receptor type 5 (CCR5), leads to homeostatic imbalance and associated diseases, necessitating therapies to regulate gene expression and production of micro-interfering ribonucleic acid (miRNA) to suppress CCR5 expression.
Innovation Solution
Compositions comprising recombinant plasmids (RPs) with nucleotide sequences encoding miRNA sequences that target and degrade CCR5 mRNA, administered via vectors like adeno-associated virus (AAV) to upregulate miRNA production, thereby reducing CCR5 levels.
Engineering Contradictions & Design Principles
Engineering Contradiction Analysis
1Quantity of substance
If miRNA production is increased to suppress CCR5 expression, then CCR5 levels are reduced, but the complexity of the therapeutic composition increases
Solution Approach 1:
The patent extracts and isolates specific miRNA sequences (e.g., miR-122, miR-146a, miR-155) that are known to target and degrade CCR5 mRNA. By selecting and extracting only the relevant miRNA sequences rather than using all possible miRNAs, the composition achieves effective CCR5 suppression while maintaining relative simplicity in design and administration.
Solution Approach 2:
The patent employs viral vectors (such as adeno-associated viruses) as intermediary carriers to deliver the miRNA-containing composition into target cells. This intermediary mechanism simplifies the delivery process and enhances the effectiveness of miRNA production without requiring direct complex intervention in the cell's transcriptional machinery.
2Quantity of substance
If recombinant plasmids are used to upregulate miRNA production, then miRNA levels increase, but the manufacturing and administration process becomes more complex
Solution Approach 1:
The patent employs viral vectors that serve multiple functions: they act as delivery vehicles, provide protective capsids, and enable efficient cellular uptake and expression of the inserted miRNA sequences. This multi-functionality simplifies the overall manufacturing process by consolidating multiple requirements into a single versatile platform rather than requiring separate systems for each function.
Solution Approach 2:
The recombinant plasmids are pre-designed and pre-assembled with the appropriate miRNA sequences and regulatory elements before administration. This preliminary preparation of the genetic construct simplifies the subsequent manufacturing and administration processes, as the complex molecular assembly is completed in advance during plasmid construction rather than during cellular production.
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 CCR5 expression by enhancing endogenous miRNA production, addressing dysregulation and potentially 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
The embodiments of the present disclosure relate to one or more compositions or methods that upregulate the production of one or more sequences of micro-interfering ribonucleic acid (miRNA). The sequences of miRNA may be complimentary to a sequence of target messenger RNA (mRNA) that encodes for translation of a target biomolecule, such as CCR5. 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. Decreasing the bioavailability of the target biomolecule within a subject that is administered the one or more compositions may address the afflictions experienced by the subject due to expression of the target biomolecule.