For the first time, scientists were able to correct the genetic mutation that causes sickle cell disease in stem cells.
In a collaborative effort, researchers at UC Berkeley, UC San Francisco Benioff Children’s Hospital Oakland Research Institute (CHORI), and the University of Utah School of Medicine fixed the mutation in modified stem cells from patients with the condition using a CRISPR/Cas9 gene editing approach.
The study, “Selection-free genome editing of the sickle mutation in human adult hematopoietic stem/progenitor cells,” was published in the journal Science Translational Medicine.
The scientists hope to re-infuse patients with the modified stem cells and alleviate disease symptoms.
“We’re very excited about the promise of this technology,” Jacob Corn, senior author on the study and scientific director of the Innovative Genomics Initiative at UC Berkeley, said in a news release. “There is still a lot of work to be done before this approach might be used in the clinic, but we’re hopeful that it will pave the way for new kinds of treatment for patients with sickle cell disease.”
The researchers observed in mice tests that after transplants, the modified stem cells stuck around for about four months, an important target of the long-lasting potential of any therapy.
“This is an important advance because for the first time we show a level of correction in stem cells that should be sufficient for a clinical benefit in persons with sickle cell anemia,” said Mark Walters, a pediatric hematologist and oncologist and director of UCSF Benioff Oakland’s Blood and Marrow Transplantation Program and co-author of the study.