Cationic Polymer Alkyl Side Chains Biomolecule Delivery
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Solution Overview
Problem
The safe and effective delivery of large molecules such as polypeptides and nucleic acids to target tissues remains a significant challenge, particularly for CRISPR systems which require the delivery of multiple components like RNA-guided endonuclease and guide RNA, posing a substantial hurdle in genetic disease treatment.
Innovation Solution
A polymer with specific structures, such as those described in Formulas 1 and 3, is developed to facilitate the delivery of nucleic acids and proteins to cells, potentially enhancing the efficacy of CRISPR systems by providing a suitable carrier mechanism.
Engineering Contradictions & Design Principles
Engineering Contradiction Analysis
1Reliability
If conventional delivery methods are used for large molecules, then delivery to target tissues is achieved, but delivery efficiency and safety are insufficient
Solution Approach 1:
The patent introduces polymeric carriers as intermediary substances that mediate between the therapeutic molecules (Cas9, guide RNA) and target cells. These polymers form complexes with the therapeutic molecules, protecting them from degradation and facilitating their uptake by target cells through endocytosis, thereby simultaneously improving delivery safety and efficiency
Solution Approach 2:
The patent employs composite polymeric structures with specific chemical compositions (including cationic polymers with alkyl side chains) that combine multiple functional properties: gene binding capability, cellular uptake promotion, and biocompatibility. These composite materials enable effective delivery of large molecules while maintaining safety profiles
2Adaptability or versatility
If multiple CRISPR components are delivered simultaneously, then complete CRISPR system functionality is achieved, but delivery complexity increases substantially
Solution Approach 1:
The patent merges multiple CRISPR components (Cas9 protein, guide RNA, and optionally donor DNA) into a single polymeric complex. The polymeric carrier simultaneously binds to all these components, allowing them to be delivered together in one formulation rather than requiring separate delivery procedures for each component
Solution Approach 2:
The polymeric delivery system is designed with universal functionality to accommodate different CRISPR components and even different CRISPR systems (e.g., Cas9, Cpf1). The same polymeric carrier can deliver various combinations of nucleic acids and proteins, reducing the need for system-specific delivery methodologies
Data Source
AI summary
Provided is a polymer comprising a structure of Formula 1 or Formula 3 as provided herein, as well as a method of making the same; a composition comprising the polymer and a nucleic acid and/or polypeptide; and a method of delivering a nucleic acid and/or polypeptide to a cell.