New SMArT platform improves safety of CRISPR gene editing

▤ Summary

Researchers at SR-Tiget developed SMArT, a new platform that improves the precision and safety of CRISPR-Cas9 editing in human blood stem cells. The system enriches correctly edited cells while filtering out cells with unwanted genomic changes such as large deletions and rearrangements. Preclinical tests showed that the selected cells engrafted successfully in mice and produced long-term human hematopoiesis. The approach could strengthen future gene therapy workflows for inherited blood and immune disorders and may fit into multiple genome engineering platforms.

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