Prime-Edited Patient Blood Stem Cells Fully Engraft and Rescue Sickle Cell Mice
New research from the labs of David Liu, PhD, at the Broad Institute, and Jonathan Yen, PhD, at St. Jude Children’s Research Hospital shows that prime editing can not only fix sickle cell disease (SCD) mutations in a patient’s hematopoietic stem and progenitor cells (HSPCs) but that these prime edited cells can also engraft and help treat genetic blood disorders in mice. These findings are among the first to establish therapeutic prime editing of HSPCs, implying that prime editing and transplanting patient HSPCs may represent a promising therapeutic strategy as a one-time autologous treatment for SCD and other blood disorders.
While allogeneic transplantation of HSPCs is the only FDA-approved treatment for SCD, most patients lack suitable donors, and the procedure is associated with serious side effects such as graft-versus-host disease and graft rejection. The use of the patient’s own HSPCs avoids immune complications and the need for a tissue-compatible donor.
There are several strategies for therapeutic manipulation of autologous SCD HSPCs currently being examined in clinical trials, and it is not yet known which strategy will be the safest and most effective for patients.
This study was published in Nature Biomedical Engineering.
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