Prime editing shows proof of concept for treating sicklecelldisease natBME nature
Scientists at St. Jude Children's Research Hospital and the Broad Institute of MIT and Harvard showed a precise genome editing approach, prime editing, can change mutated hemoglobin genes back to their normal form in SCD patient cells, which restores normal blood parameters after transplantation into mice. The findings were published today inScientists have rapidly developed technologies to edit DNA, including Cas9 nucleases and base editors, to treat genetic diseases.
"These results show efficient prime editing of blood stem cells and that the prime-edited cells maintain their full ability to engraft and repopulate the bone marrow of an animal," said senior and co-corresponding author David Liu, Ph.D., Richard Merkin, Professor at Broad Institute of MIT and Harvard, whose lab invented prime editing in 2019.
While the scientists conducted the research in SCD patient cells transplanted into mice, the approach may have advantages over current genome editing methods used in clinical trials, such as Cas9 nucleases, which make double-stranded breaks in DNA that prime editing largely avoids. The collaborators had previously shown base editing, an alternative genome editing technology, could turn the sickle cell mutation into a benign variant, but not the original healthy sequence, in a 2021publication.
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