Scientists are inching closer to developing single-shot gene editing for blood disorders.
What is the most effective method to repair defective blood cells in the largest number of people? A swarm of gene editing researchers are focusing on this issue. A new article in Science presents an idea.
Researchers loaded lipid nanoparticles, the small clusters of fat, with cellular instruction manuals in the form of mRNA—tthe same fundamental design as the COVID mRNA vaccines. Antibodies on the surface of these therapeutic bundles direct them to the hematopoietic progenitor cells in the bone marrow. Once there, they delivered mRNA-containing gene-editing technology and corrected sickle cell defects in both mice and human cells.
Paula Cannon, a gene therapy researcher at the Keck School of Medicine of the University of Southern California who was not involved in the paper, remarked, “What really struck me was how efficient it is.”