CRISPR-Cas9 Delivery via Segmented Viral and Non-Viral Vectors
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Solution Overview
Problem
There is a need for safe and effective means of delivering gene editing tools, such as CRISPR-Cas9, to cells for therapeutic applications, particularly for in vivo and ex vivo gene editing, as existing methods face challenges in achieving efficient and targeted nucleotide sequence modification.
Innovation Solution
The use of delivery systems comprising guide RNA (gRNA) and nucleic acid editing systems, including CRISPR-Cas9, in combination with repair templates, administered via viral and non-viral vectors like lipid nanoparticles and AAV vectors, to facilitate efficient in vivo and ex vivo gene editing, with options for temporal and tissue-specific expression.
Engineering Contradictions & Design Principles
Engineering Contradiction Analysis
1Reliability
If gene editing tools are delivered to cells for therapeutic applications, then genetic diseases can be corrected, but delivery safety and efficiency become challenging
Solution Approach 1:
The patent segments the gene editing system into separate components (guide RNA and nucleic acid editing system) that can be delivered independently via different vehicles. This allows optimization of each component's delivery route and reduces the risk associated with delivering the complete system simultaneously, thereby improving safety while maintaining editing efficacy.
Solution Approach 2:
The patent employs delivery vehicles (viral and non-viral vectors) as intermediaries to transport the gene editing components to target cells. These intermediaries protect the editing tools during transit, control their release, and reduce direct exposure risks, thus enhancing delivery safety while ensuring the editing system reaches its destination effectively.
2Adaptability or versatility
If multiple delivery vehicles are used to deliver gRNA and nucleic acid editing system, then delivery flexibility increases, but system complexity increases
Solution Approach 1:
The patent employs both viral and non-viral delivery vehicles that can be used across different gene editing applications and target cell types. These multi-functional vehicles can deliver various nucleic acid components (gRNA, editing systems, repair templates) through standardized protocols, providing delivery flexibility without proportionally increasing system complexity.
3Manufacturing precision
If repair templates are included in the delivery system, then gene editing precision improves, but delivery complexity increases
Solution Approach 1:
The patent merges the repair template delivery with the existing delivery system for gRNA and nucleic acid editing components. By combining multiple functions (editing + repair) into a unified delivery approach using the same viral or non-viral vectors, the system achieves precise nucleotide modification without proportionally increasing delivery complexity.
Data Source
AI summary
The present disclosure relates to compositions and methods for modifying a gene sequence, and for systems for delivering such compositions. For example, the disclosure relates to modifying a gene sequence using a CRISPR-Cas9 or other nucleic acid editing system, and methods and delivery systems for achieving such gene modification, such as viral or non-viral delivery systems.