A study using a CRISPR-based approach to treat sickle-cell disease has reported promising results, the Associated Press reports.
Last year, Vertex Pharmaceuticals and CRISPR Therapeutics launched a study in which doctors isolated cells from patients' bone marrow and edited them to produce the fetal version of hemoglobin, before re-introducing the cells to patients. About a year after treatment, study participant Victoria Gray told NPR she was doing well and was producing fetal hemoglobin.
The AP notes additional data from the study have now been presented at the American Society of Hematology conference and published in the New England Journal of Medicine. In NEJM, Sarah Cannon Center for Blood Cancer's Haydar Frangoul and his colleagues report that two patients from their study, one with sickle-cell disease and one with β-thalassemia, have had sustained increases in fetal hemoglobin levels. Frangoul and his colleagues note they have since treated a further eight patients with similar results.
"The preliminary results are extremely encouraging," Frangoul tells the AP.
In a separate NEJM paper, researchers from Harvard Medical School report on their study of six patients with severe sickle cell disease who they treated using a shRNA targeting the same gene. They too report that their patients had increased fetal hemoglobin levels.