Nucleic Acid Complexes via Cyclodextrin Host-Guest Condensation
Find Innovative SolutionsGenerate Solutions
Solution Overview
Problem
Current methods for delivering nucleic acids, such as siRNA, into cells face challenges with toxicity and efficiency, particularly with viral vectors due to immunogenicity and scalability issues, while non-viral vectors lack efficacy.
Innovation Solution
A nucleic acid complex comprising a nucleic acid, a macrocyclic compound, and a pendant polymer modified with a hydrophobic group, forming a host:guest polymer complex, which facilitates safe and efficient delivery by condensing the nucleic acid into nanoparticles for intracellular uptake.
Engineering Contradictions & Design Principles
Engineering Contradiction Analysis
1Productivity
If viral vectors are used for nucleic acid delivery, then delivery efficiency is improved, but immunogenicity and safety issues worsen
Solution Approach 1:
The patent uses a non-viral vector system comprising a cationic polymer and cyclodextrin complex as an intermediary to deliver nucleic acids without the immunogenicity of viral vectors, while achieving comparable delivery efficiency through endosomal escape mechanisms
Solution Approach 2:
The patent modifies the chemical parameters of the delivery system by using specific cationic polymers with controlled molecular weight and charge density, combined with cyclodextrin host-guest complexes, to optimize both delivery efficiency and reduce immunogenicity
2Reliability
If non-viral vectors are used for nucleic acid delivery, then safety and scalability are improved, but delivery efficiency worsens
Solution Approach 1:
The patent creates a composite delivery system combining cationic polymers with cyclodextrin molecules, where the polymer provides safety and biocompatibility while the cyclodextrin component enables efficient endosomal escape, achieving both safety and delivery efficiency
Solution Approach 2:
The patent employs a nested structure where nucleic acids are complexed within cyclodextrin cavities, which are in turn associated with cationic polymer chains, creating multiple protective and functional layers that enhance both safety and delivery efficiency
3Measurement precision
If siRNA is delivered to achieve gene knockdown, then therapeutic specificity is improved, but delivery efficiency and cell uptake worsen
Solution Approach 1:
The patent applies local quality by designing the delivery system with specific functional regions: the cationic polymer provides overall structural integrity and cell interaction, while cyclodextrin units at specific locations provide siRNA binding and endosomal escape functionality, optimizing both specificity and delivery
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 nucleic acid complex achieves high gene knockdown efficiency with low cytotoxicity, comparable to conventional cationic polymer transfection agents, and effectively delivers siRNA into cells, promoting endosomal escape and silencing of disease-related genes.
Implementation Method 1
wherein the macrocyclic compound and the pendant polymer form a host:guest polymer complex
Implementation Method 2
wherein the pendant polymer is modified with a hydrophobic group
Data Source
AI summary
The invention relates to nucleic acid complexes, methods of preparation thereof, and uses thereof for delivering a nucleic acid into a cell.