Professional technology integration to support the entire R&D process

Off-target effects occur when gene editing tools (such as CRISPR-Cas9) accidentally identify and cleave other similar, but not identical, sequences in the genome while targeting and cleaving a specific target DNA sequence. These unintended cleavages can lead to gene mutations, cellular dysfunction, and even cancer, and are one of the most significant safety concerns in gene therapy and clinical applications.

The core principle of off-target effect analysis is to comprehensively screen the edited genome for unintended damage through a combination of bioinformatics prediction and experimental verification.

Off-target effect analysis is crucial and primarily focuses on:

1. Basic Research: Ensuring the accuracy of gene function research results to avoid misinterpreting phenotypes caused by off-target effects as the function of the target gene.

2. Drug Development and Cell Therapy: In the development of engineered cell therapies such as CAR-T, it is imperative to ensure the accuracy of editing to prevent off-target mutations from causing cell cancer or dysfunction.

3. Clinical Gene Therapy: This is the bottom line of safety. Any clinical trial based on gene editing must provide rigorous off-target analysis data to maximize patient safety. 4. Agricultural Breeding: When developing genetically modified crops, off-target effects must be eliminated to ensure genetic stability and environmental safety.

Directly identifying and assessing potential risks is the cornerstone for advancing gene editing technology toward clinical application.
Ensuring Data Reliability
In scientific research, it can effectively avoid false positives in experimental results, enhancing the rigor and reproducibility of research.
Guiding Tool Optimization
Analysis results can be used to inform researchers and design more specific and high-fidelity gene editing tools (such as high-fidelity Cas9 variants), thereby reducing off-target effects at the source.