Mesoporous Silica Nanoparticles with Expanded Pores for Therapeutic Delivery
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Solution Overview
Problem
Current delivery methods for therapeutic agents, such as gene-editing agents, face challenges in achieving efficacy in vivo due to inefficiencies in delivery, particularly for agents that are effective in cellular assays but show reduced performance when administered systemically.
Innovation Solution
A construct comprising a porous core with a cargo and a spacer, where the core has pores large enough to accommodate the cargo, and an outer layer of lipid or polymer, facilitating targeted delivery by forming a construct with a mesoporous silica nanoparticle core, a spacer for binding the cargo, and a lipid or polymer layer for enhanced cellular uptake.
Engineering Contradictions & Design Principles
Engineering Contradiction Analysis
1Reliability
If traditional delivery methods are used for gene-editing agents, then the agents can be administered systemically, but delivery efficiency is low and in vivo efficacy is reduced
Solution Approach 1:
The patent employs mesoporous silica nanoparticles with controlled pore sizes (5-35 nm) that can accommodate large gene-editing cargo molecules. The porous structure allows efficient loading of therapeutic agents while maintaining particle stability for systemic administration, directly resolving the contradiction between delivery efficiency and in vivo efficacy
Solution Approach 2:
The invention creates a composite delivery system combining mesoporous silica core with surface-modified lipids or polymers. This composite structure integrates the cargo-loading capability of porous silica with the cellular uptake enhancement of lipid/polymer coatings, thereby improving both delivery efficiency and in vivo efficacy simultaneously
2Quantity of substance
If pore size is increased to accommodate large cargo, then cargo loading capacity improves, but particle stability may be compromised
Solution Approach 1:
The patent systematically varies pore size parameters (5-35 nm range) to optimize the balance between cargo accommodation and particle stability. By controlling pore dimensions within this specific range, the system achieves sufficient loading capacity for large gene-editing agents while maintaining structural integrity for systemic circulation
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 construct enables efficient delivery of therapeutic agents, including CRISPR components, by expanding the pores of the core to accommodate the cargo and forming a lipid layer for improved cellular uptake, thereby enhancing the in vivo efficacy of therapeutic agents.
Implementation Method 1
expanding the pores of the core to accommodate the cargo
Implementation Method 2
a spacer for binding the cargo
Implementation Method 3
a lipid or polymer layer for enhanced cellular uptake
Data Source
AI summary
The present invention relates to a construct including a porous core, a cargo, and a spacer disposed between the core and the cargo. In some examples, the construct further includes an outer layer composed of a lipid, a polymer, or a combination thereof. Methods of making and employing such constructs are also described herein.