By Natasha Bhatt / May 27, 2024
CRISPR is a powerful tool for editing genomes which is the process of splitting the genome into segments and then piecing it back together, however, it has an inherent danger. CRISPR gene editing causes large and unpredictable DNA deletions thus causing unintended consequences to the cells. Researchers at KAUST have created a novel method to improve CRISPR gene editing's accuracy and safety. This technique tackles the important issue of large scale DNA deletion that is highlighted by the genome-slicing and reconnection mechanisms of CRISPR.
A group, led by stem cell researcher Mo Li, identified the function of the repair process known as microhomology-mediated end joining(MMEJ) that causes significant losses and results in large deletions of the nucleotides.
The study highlights two genes: RPA which aids in genome protection, the POLQ, which raises the likelihood of significant deletions. Through genetic techniques or drug inhibition of POLQ, the researchers increased RPA expression and decreased harmful deletions without compromising editing efficiency. Additionally, by enhancing homology-directed repair, the inventions ensured accurate genome editing.
Using this approach, an experiment led on stem cells that employ sickle cell disease and Wiskott-Aldrich syndrome-related mutations demonstrates that this method of gene editing is very accurate and dependable. This approach represents a major advance in CRISPR technology by moving towards a safer and more effective genetic treatment option available. Mo Li emphasizes the excitement around these findings as he states, "it’s really exciting because it means we’re getting closer to safer and more effective treatments for genetic diseases.”
Citations:
King Abdullah University of Science & Technology (KAUST). (2024, May 26). Simple new strategy improves safety and precision of CRISPR gene editing. SciTechDaily. https://scitechdaily.com/simple-new-strategy-improves-safety-and-precision-of-crispr-gene-editing/
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