A University of California, Berkeley, team of researchers has designed a CRISPR-Cas9-based gene editing approach to potentially treat sickle cell disease, the Los Angeles Times reports.
"What we have right now, if we can scale it up and make sure it works well, is already enough to form the basis of a clinical trial to cure sickle cell disease with gene editing," first author Mark DeWitt, a postdoc at Berkeley's Innovative Genomics Initiative, tells the LA Times.
De Witt and his colleagues obtained hematopoietic stem cells from sickle cell patients and designed a ribonucleoprotein complex to replace the sickle cell disease mutation in those cells. As the team reports in Science Translational Medicine, when in culture, these corrected cells produced less sickled hemoglobin and increased amounts of wild-type hemoglobin. Only a small percentage of cells were fully fixed, the Verge notes, but it adds that while that's not "ideal," it could be enough to give some relief to patients.
In addition, when the Berkeley team transferred edited human blood stem cells into the bone marrow of five mice, these gene edits lasted through 16 weeks. However, the LA Times notes just 2.3 percent of cells contained edited DNA.
"It will be years of very careful and rigorous study to ensure that it's totally and completely safe before we even contemplate an actual clinical trial," DeWitt adds. "But we have a path. We just have to walk down it."