Each year an estimated 8 million children are born worldwide with severe genetic disorders. During pregnancy, genetic disorders can impair fetal development and lead to birth defects, but currently no approved medical treatments exist to correct gene mutations before birth.
Now, in a study published on June 26 in Nature Communications, researchers from Carnegie Mellon and Yale universities used a unique gene modification method to correct gene mutations in mice early in embryonic development.
Some scientists have used the popular CRISPR-Cas9 and similar methods to attempt in utero gene modification. These techniques use enzymes to cut out DNA mutations but run the risk of cutting other, unintended areas of DNA. Such accidental edits in embryos can cause severe genetic damage that future offspring could inherit.
In the new study, the research team used a method of gene editing that does not involve cutting DNA. The technique binds synthetic DNA to mutation sites, spurring the cell’s own natural repair pathways into action to correct the error. The researchers used the technique to treat mouse embryos that carried a genetic mutation known to cause beta thalassemia, an inherited blood disorder. (Beta thalassemia impairs the body’s ability to make hemoglobin, a protein that carries oxygen to every cell in the body.)
After only a single treatment in utero, the mice were born with normal hemoglobin levels and researchers considered them cured. The animals showed no evidence of editing errors. “We looked at 50 million samples and couldn’t find one offsite error when we used our … gene editing technique,” Danith Ly, one of the researchers, said in a statement.
David Prentice, a biochemist and vice president of the pro-life Charlotte Lozier Institute, believes the research might offer a safe and accurate way to treat genetic diseases before they cause damage. “We aren’t talking about designer babies or any kind of germ-line editing where we are manufacturing embryos,” he told me. “This is an ethical way to treat these diseases and get at them even before the baby is born.”
Brown plant hoppers, major insect pests on rice plants in Asia, somehow know when it’s time to grow longer wings and fly away to greener pastures. Research published in the July 17 issue of Proceedings of the National Academy of Sciences suggests the insects get their marching orders from the rice plants themselves.