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Precise genome-editing approaches can correct mutations that cause sickle cell disease

Precise genome-editing approaches can correct mutations that cause sickle cell disease

 


Improving the accuracy of gene editing

“We have identified what could be the next wave of hereditary anemia treatments,” said co-author Mitchell Weiss, MD, Ph.D., St. Jude Department of Hematology chair. “We have taken the latest state-of-the-art genetic engineering technology and demonstrated that meaningful gene editing is possible for future treatments.”

The scientists worked with SCD patient cells transplanted into mice, an approach that has been used in clinical trials, including the Cas9 nuclease, which makes double-strand breaks in DNA that prime editing largely evades. It may be superior to current genome editing methods. Collaborators have previously demonstrated base editing, an alternative genome-editing technique that can turn sickle cell disease mutations into benign variants, but not the original healthy sequences, in 2021. rice field. Nature Publishing. The current study showed that prime editing can turn disease mutations back into the normal gene variant through a T to A conversion that is not possible with base editing.

This study showed the potential benefits of using Prime Editing to cure hereditary anemia, but it also showed the limitations. requires a time-consuming process of For example, targeting the prime-editing system to the appropriate DNA regions and designing prime-editing guide RNAs (pegRNAs) that specify the desired edits.

safety first

Safety remains a concern for all genome editing technologies, especially newer approaches. Consistent with reports from other labs on prime-editing, the current study showed that off-target prime-editing was virtually non-existent, but could pose unforeseen safety issues as a new gene-editing technique. there is.

“We do our best to predict toxicity, but we don’t know the true extent of the risks of this treatment until it’s used in a patient.

Even with these challenges, scientists are optimistic about the future of prime editing.

“The unique versatility of Prime Editing has the potential to cure even more genetic diseases,” Yen said. “It would be hard to go to the clinic. It would require extensive manufacturing development, process optimization and safety evaluation. But the proof of concept is there. It is opening the door to the development of therapeutics for

author and funding

The study’s first author was Kelsey Everett, a graduate student in Liu’s lab at the Broad Institute, who joined the St. Jude Collaborative Research Consortium for Sickle Cell Disease to advance the study. Other authors are Rachel Levine, Karin Mayberry, Yunjung Jang, Tyagaraj Mayuranathan, Nikita Nimagadda, Erin Dempsey, Yichao Lee, Senthil Bhuparan, and Yong Cheng, all from St. Jude. Gregory Newby, Jessie Davis, Andrew Nelson, Peter Chen, Alexander Sousa (Broad Institute). Xiong Liu and John Tisdale, National Heart, Lung, and Blood Institute, National Institute of Diabetes and Digestive and Kidney Diseases.

This study was supported by the National Institutes of Health (U01 AI142756, RM1 HG009490, R35 GM118062, R01 HL156647, R01 HL136135, P01 HL053749, P30 CA21765), Bill and Melinda Gates Foundation, Howard Hughes Medical Institute (Helen Hay Whitney Postdoctoral Fellowship), St. Jude Collaborative Research Consortium for Sickle Cell Disease, National Science Foundation GRFP Fellowship, ALSAC, St. Jude’s fundraising and advocacy organization.

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