Cell-Penetrating Peptides for Genome-Editing Delivery
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Solution Overview
Problem
Current methods for delivering genome-editing system molecules, such as CRISPR system molecules, face challenges in efficiency and safety due to limitations in existing delivery vehicles like viral vectors and chemical transfection techniques.
Innovation Solution
The development of complexes and nanoparticles comprising cell-penetrating peptides (CPPs) that stabilize and deliver genome-editing system molecules, including CRISPR-associated proteins and nucleic acids, into cells, using CPPs like VEPEP-3, VEPEP-6, VEPEP-9, and ADGN-100 peptides to form stable complexes capable of efficient cellular uptake.
Engineering Contradictions & Design Principles
Engineering Contradiction Analysis
1Productivity
If viral vectors are used for delivering genome-editing molecules, then delivery efficiency is improved, but safety and complexity worsen
Solution Approach 1:
The patent uses cell-penetrating peptides as intermediary carriers to deliver genome-editing molecules into cells. These peptides act as mediators that facilitate cellular uptake without the safety concerns of viral vectors, resolving the contradiction between delivery efficiency and safety by providing a non-viral alternative that maintains effectiveness while improving reliability
Solution Approach 2:
The patent extracts and utilizes only the essential cell-penetrating function from viral vectors by employing synthetic cell-penetrating peptides. This extraction approach removes the harmful viral components while retaining the beneficial delivery mechanism, thereby improving safety while maintaining delivery efficiency
2Productivity
If chemical transfection techniques are used for delivering genome-editing molecules, then delivery capability is achieved, but toxicity and complexity increase
Solution Approach 1:
The patent changes the chemical parameters of delivery vehicles by using cell-penetrating peptides with specific amino acid sequences and properties. These parameter changes result in lower toxicity compared to traditional chemical transfection reagents while maintaining delivery capability, as the peptides are biocompatible and can be cleared more easily by biological systems
Solution Approach 2:
The patent employs composite structures formed by cell-penetrating peptides complexing with genome-editing molecules. This composite approach creates a delivery system that combines the benefits of peptide-mediated cellular uptake with the therapeutic function of genome-editing molecules, reducing toxicity while achieving effective delivery
3Productivity
If existing delivery vehicles are used, then genome-editing molecules can be delivered, but stability and safety worsen
Solution Approach 1:
The patent applies preliminary stabilization measures by forming pre-complexes between cell-penetrating peptides and genome-editing molecules before cellular uptake. This preliminary action protects the genome-editing molecules from degradation during delivery, improving stability while maintaining delivery capability
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
This approach enables efficient and safer delivery of genome-editing molecules into cells, overcoming the limitations of existing methods by using less complex, less toxic, and more effective CPP-based delivery systems, which can effectively modify target polynucleotides for therapeutic applications.
Implementation Method 1
cell-penetrating peptides (CPPs) that stabilize and deliver genome-editing system molecules, including CRISPR-associated proteins and nucleic acids, into cells
Data Source
Figure 1A~1B
Figure 2A~2D
Figure 3A~3D
AI summary
The present invention pertains to peptide-containing complexes/nanoparticles that are useful for stabilizing and/or delivering one or more molecules of a genome-editing system, such as proteins and/or nucleic acids, for example CRISPR proteins and/or nucleic acids.