Two trials show promising results with gene therapies that target sickle-cell anemia




Two teams of researchers working independently have found success in trialing gene therapies targeting sickle-cell anemia. Both teams have published papers in the New England Journal of Medicine describing their work and results. The first team comprised members from the U.S., Germany, Canada and France; they used the CRISPR-Cas9 gene editing system to boost the production of fetal hemoglobin in sickle-cell anemia patients. The second team comprised members from Harvard Medical School and the Dana–Farber Cancer Institute. They also sought to boost the production of fetal hemoglobin in sickle-cell anemia patients but used a different technique that involved introducing RNA via a viral carrier that altered expression of the fetal hemoglobin gene.



MedicalXpress