Sickle cell disease (SCD) is a perfect candidate for gene editing…Correcting the mutation behind SCD is an obvious strategy. How to do so is where creativity comes in.
…
1. Researchers at Johns Hopkins edited one copy of the mutant beta globin gene in iPS cells from patients, leading some red blood cells to make normal hemoglobin….
…
2. Trimming an “enhancer” gene (BCL11A) dialed down the molecular switch from fetal to adult hemoglobin, enabling some of the functioning fetal version to persist, and compensate for sickled adult beta globin.
It was an indirect approach. “Our goal was to break the enhancer, rather than fix the hemoglobin mutation…” said…Daniel Bauer, MD, PhD, a pediatric hematologist/oncologist at Dana-Farber/Boston Children’s.
…
3. Mark DeWitt, of the University of California, Berkeley and co-workers describe experiments that in a way combine the two above: they edited the beta globin gene in HSCs from SCD patients. Instead of delivering the correct DNA sequence aboard viruses or liposomes, they used a single guide RNA along with single-stranded pieces of DNA to target the beta globin gene.
The GLP aggregated and excerpted this blog/article to reflect the diversity of news, opinion, and analysis. Read full, original post: 3 Gene Editing Approaches for Sickle Cell Disease
