Lipid Nanoparticles for Gene Editing Delivery

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Solution Overview

Problem

Current gene-editing technologies, such as CRISPR-Cas9, face challenges with low editing efficiency, off-target site cleavages, and inefficient delivery of gene-editing tools into target cells, which can lead to cell death and genomic integrity issues.

Innovation Solution

The development of cell-permeable, multi-functionalized nanoparticles that are covalently linked with bioactive molecules, allowing for high specificity and efficiency in penetrating cell membranes, binding target genes, and introducing gene-editing modifications.

Engineering Contradictions & Design Principles

VSEngineering Contradiction Analysis

1Reliability

If CRISPR-Cas9 systems are used for gene editing, then gene editing capability is achieved, but delivery efficiency into target cells is low and off-target cleavages occur

Engineering Contradiction:
Improvegene editing accuracyVSAvoiddelivery efficiency
Core Design Contradiction:
ReliabilityVSProductivity

Solution Approach 1:

The patent uses lipid nanoparticles as intermediary carriers to deliver CRISPR-Cas9 components into target cells. The lipid nanoparticle formulation enables efficient cellular uptake while protecting the genetic material, thereby improving delivery efficiency without compromising editing accuracy

Inventive Principle:
Principle #24Intermediary (Mediator)

Solution Approach 2:

The patent optimizes multiple parameters including lipid composition, nanoparticle size, charge, and RNA-to-lipid ratios to enhance delivery efficiency. By adjusting these physical and chemical parameters, the system achieves high transfection efficiency while maintaining specificity and reducing off-target effects

Inventive Principle:
Principle #35Parameter changes

2Productivity

If gene editing tools are delivered into target cells, then gene editing efficiency is improved, but cell death and genomic integrity issues increase

Engineering Contradiction:
Improvegene editing efficiencyVSAvoidcell death and genomic damage
Core Design Contradiction:
ProductivityVSObject-affected harmful factors

Solution Approach 1:

The patent employs transient expression of Cas9 protein and guide RNA delivered via lipid nanoparticles, which are naturally degraded after performing their editing function. This transient approach avoids long-term presence of editing components, reducing off-target effects and genotoxicity while maintaining high editing efficiency

Inventive Principle:
Principle #27Cheap short-living objects (Disposable)

Solution Approach 2:

The lipid nanoparticle formulation provides protective cushioning for the CRISPR components during cellular entry, and the use of optimized delivery conditions minimizes cellular stress and toxicity before the editing process begins, thereby protecting cell viability

Inventive Principle:
Principle #11Beforehand cushioning (Prior cushioning)

3Ease of operation

If traditional gene delivery methods are used, then delivery process is simple, but specificity and efficiency of target gene binding are low

Engineering Contradiction:
Improvedelivery process simplicityVSAvoidtarget gene binding specificity
Core Design Contradiction:
Ease of operationVSManufacturing precision

Solution Approach 1:

The lipid nanoparticle platform serves multiple functions simultaneously: it protects genetic material, facilitates cellular entry, enables cytoplasmic delivery, and can be tailored for specific target genes through guide RNA design. This multi-functional approach maintains operational simplicity while achieving high specificity

Inventive Principle:
Principle #6Universality (Multi-functionality)

Data Source

PatentUS20250101425A1Nanoparticles functionalized with gene editing tools and related methods
Publication Date: 2025.03.27 STEMGENICS INC

AI summary

This disclosure relates to compositions and methods for editing or altering target nucleotide sequences based on nanoparticle delivery vehicles. The compositions and methods can be applied to influence the functional expression of target gene products encoded by DNA and/or RNA. In some embodiments, the altered gene sequences are useful to normalize and regulate the function of target cells.