RNA-Protein Complexes for Enhanced Gene Delivery
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Solution Overview
Problem
Current methods for delivering biologically active RNA molecules to cells face low transfection efficiencies, limiting their therapeutic potential, especially in vivo where achieving uniform gene regulation across a population of cells is difficult.
Innovation Solution
The development of bioreactor cells that produce and secrete RNA-protein complexes, comprising biologically active RNA molecules and fusion proteins with RNA binding domains and transport peptides, allowing for the delivery of these molecules to the extracellular matrix and neighboring cells, thereby amplifying the modulatory signal beyond initial transfection limits.
Engineering Contradictions & Design Principles
Engineering Contradiction Analysis
1Productivity
If traditional transfection methods are used to deliver biologically active RNA molecules to cells, then the delivery process is simple, but the transfection efficiency is low
Solution Approach 1:
The patent employs RNA-binding proteins as intermediary carriers that selectively bind to biologically active RNA molecules (such as siRNA, miRNA, or shRNA) and facilitate their delivery into target cells. These protein-RNA complexes act as mediators that overcome the low transfection efficiency of traditional methods by providing a protected, targeted delivery mechanism that enhances cellular uptake and intracellular delivery of the RNA therapeutics.
2Productivity
If bioreactor cells are used to produce and secrete RNA-protein complexes, then the delivery efficacy is enhanced, but the system complexity increases
Solution Approach 1:
The patent utilizes bioreactor cells that are engineered to autonomously produce, process, and secrete RNA-protein complexes without requiring external intervention. The bioreactor cells perform the entire delivery function through their own cellular machinery, transforming them into self-sufficient therapeutic delivery systems that continuously generate and release active RNA complexes to target cells in the extracellular matrix.
Solution Approach 2:
The patent employs RNA-binding proteins as intermediary carriers that selectively bind to biologically active RNA molecules (such as siRNA, miRNA, or shRNA) and facilitate their delivery into target cells. These protein-RNA complexes act as mediators that overcome the low transfection efficiency of traditional methods by providing a protected, targeted delivery mechanism that enhances cellular uptake and intracellular delivery of the RNA therapeutics.
Data Source
AI summary
The present invention provides novel compounds, compositions, and methods for the delivery of biologically active RNA molecules to cells. Specifically, the invention provides novel nucleic acid molecules, polypeptides, and RNA-protein complexes useful for the delivery of biologically active RNAs to cells and polynucleotides encoding the same. The invention also provides vectors for expressing said polynucleotides. In addition, the invention provides cells and compositions comprising the novel compounds and vectors, which can be used as transfection reagents. The invention further provides methods for producing said compounds, vectors, cells, and compositions. Additionally, vectors and methods for delivering biologically active RNA molecules to cells and/or tissues are provided. The novel compounds, vectors, cells, and compositions are useful, for example, in delivering biologically active RNA molecules to cells to modulate target gene expression in the diagnosis, prevention, amelioration, and/or treatment of diseases, disorders, or conditions in a subject or organism.