Sumary of Gene Editing Technique Corrects Sickle Cell Disease in Mice:
- The investigators reported using genetic-based editing on mice to convert a disease-causing hemoglobin gene to a benign variant that would enable healthy blood cell production..
- The root of SCD is two mutated copies of the hemoglobin gene, HBB, which cause red blood cells to transform from a circular disc into a sickle shape..
- Jude Children’s Research Hospital used a molecular technology called base editing to directly convert a single letter of the disease-causing DNA into a harmless genetic variant of HBB in human blood-producing cells and in a mouse model of SCD..
- “We were able to correct the disease-causing variant in both cell and animal models using a customized base editor, without requiring double-stranded DNA breaks or inserting new segments of DNA into the genome,”.
- explained co-senior author David Liu, director of the Merkin Institute of Transformative Technologies in Healthcare at the Broad Institute, a Harvard professor, and a Howard Hughes Medical Institute investigator..
- “In particular, we combined expertise in protein engineering, base editing and red blood cell biology to create a novel approach for treating and possibly curing sickle cell disease,”…