OMNI-103 CRISPR Nuclease RNA Complex Specificity

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Solution Overview

Problem

Current CRISPR systems face limitations in sequence specificity, expression, and delivery, which restrict their applicability in genome engineering and editing.

Innovation Solution

A composition comprising a non-naturally occurring RNA molecule that forms a complex with and targets an OMNI-103 CRISPR nuclease to a specific DNA target site, utilizing a crRNA repeat sequence portion and a guide sequence portion, in the presence of a tracrRNA sequence.

Engineering Contradictions & Design Principles

VSEngineering Contradiction Analysis

1Measurement precision

If conventional CRISPR systems are used for genome editing, then basic genome editing capability is achieved, but sequence specificity and delivery efficiency are limited

Engineering Contradiction:

Improvesequence specificityVSAvoidapplicability in genome engineering

Core Design Contradiction:

Measurement precisionVSAdaptability or versatility

Solution Approach 1:

The CRISPR system is divided into distinct functional modules: a customized guide RNA (gRNA) component for sequence-specific targeting, an OMNI-103 nuclease component for DNA cleavage, and optional delivery vehicle components. This segmentation allows independent optimization of each module's function, with the gRNA providing high sequence specificity while the nuclease provides efficient cleavage, and delivery vehicles addressing transport limitations.

Inventive Principle:

Principle #1Segmentation

Solution Approach 2:

The OMNI-103 CRISPR nuclease system is designed with universal applicability across multiple genome engineering applications. The same core nuclease-gRNA complex can be applied to gene knockout, gene activation, base editing, and diagnostic applications by simply changing the gRNA sequence, making the system versatile while maintaining high sequence specificity through the programmable gRNA component.

Inventive Principle:

Principle #6Universality (Multi-functionality)

2Productivity

If CRISPR nucleases are delivered to target cells, then genome editing is achieved, but delivery challenges and expression limitations restrict applicability

Engineering Contradiction:

Improvegenome editing efficiencyVSAvoiddelivery and expression

Core Design Contradiction:

ProductivityVSEase of operation

Solution Approach 1:

The system employs intermediary delivery vehicles such as liposomes, viral vectors, or electroporation reagents that mediate the transport of the CRISPR nuclease and gRNA complex into target cells. These intermediaries protect the genetic material during delivery, facilitate cellular uptake, and enable efficient expression of the CRISPR components, thereby overcoming direct delivery challenges while maintaining high genome editing efficiency.

Inventive Principle:

Principle #24Intermediary (Mediator)

3Adaptability or versatility

If different CRISPR nucleases are used to target various genomic loci, then broader targeting capability is achieved, but pre-immunity and specificity limitations arise

Engineering Contradiction:

Improvetargeting capabilityVSAvoidspecificity and pre-immunity

Core Design Contradiction:

Adaptability or versatilityVSReliability

Solution Approach 1:

The system achieves different targeting capabilities by modifying only the local gRNA sequence component while keeping the core OMNI-103 nuclease unchanged. Each specific genomic locus is targeted by a custom-designed gRNA with sequence complementarity to that specific target, providing localized adaptation without affecting the overall reliability and specificity of the nuclease enzyme. This approach eliminates pre-immunity issues associated with repeated use of the same nuclease.

Inventive Principle:

Principle #3Local quality

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

Enables efficient genome editing, including double-strand breaks at predetermined target sites, allowing for mutation, insertion, and deletion of DNA sequences, thereby facilitating genomic engineering and diagnostics.

Implementation Method 1

the RNA molecule comprises a crRNA repeat sequence portion and a guide sequence portion, wherein the RNA molecule forms a complex with and targets an OMNI-103 CRISPR nuclease to a DNA target site

Methodology Applied

Scientific EffectBase pairing:

Implementation Method 2

the RNA molecule forms a complex with and targets an OMNI-103 CRISPR nuclease to a DNA target site having complementarity to a guide sequence portion

Methodology Applied

Scientific EffectMolecular recognition:

Implementation Method 3

enables efficient genome editing, including double-strand breaks at predetermined target sites

Methodology Applied

Scientific EffectNuclease cleavage: Enzyme

Data Source

PatentUS20250034596A1Omni-103 CRISPR Nuclease-RNA Complexes

Publication Date: 2025.01.30 EMENDOBIO INC

AI summary

A composition comprising a non-naturally occurring RNA molecule, the RNA molecule comprising an RNA scaffold portion, the RNA scaffold portion having the structure: crRNA repeat sequence portion-tracrRNA portion: wherein the RNA scaffold portion forms a complex with and targets an OMNI-103 CRISPR nuclease to a DNA target site having complementarity to a guide sequence portion of the RNA molecule.