Article
Ex vivo prime editing of patient haematopoietic stem cells rescues sickle-cell disease phenotypes after engraftment in mice.
Nature biomedical engineering - 1 May 2023
Everette Kelcee A, Newby Gregory A, Levine Rachel M, Mayberry Kalin, Jang Yoonjeong, Mayuranathan Thiyagaraj, Nimmagadda Nikitha, Dempsey Erin, Li Yichao, Bhoopalan Senthil Velan, Liu Xiong, Davis Jessie R, Nelson Andrew T, Chen Peter J, Sousa Alexander A, Cheng Yong, Tisdale John F, Weiss Mitchell J, Yen Jonathan S, Liu David R
Abstract excerpt
Sickle-cell disease (SCD) is caused by an A·T-to-T·A transversion mutation in the β-globin gene (HBB). Here we show that prime editing can correct the SCD allele (HBBS) to wild type (HBBA) at frequencies of 15%-41% in haematopoietic stem and progenitor cells (HSPCs) from patients with SCD. Seventeen weeks after transplantation into immunodeficient mice, prime-edited SCD HSPCs maintained HBBA levels and displayed...
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