Scientists Successfully Utilize Gene Editing to Remove Fatal Blood Disorder

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Beta- thalassemia, a blood disorder that plagues individuals throughout the entirety of their lives. beta-thalassemia has no viable cure and the only real hope that people have of overcoming this disease is either a stem cell or bone marrow transplant. However, the procedures for beta-thalassemia are rarely performed due to the life-threatening risk that comes with them.

With limited options, people who suffer from beta-thalassemia will need to have lifelong blood transfusions and specialist care. The disease is genetic, children who contract it from their parents may develop life-threatening anemia, blood clots, misshapen bones, jaundice and many other issues. However, there is new hope in the form of genetic engineering.

A research team from Sun Yatsen University have used gene editing to correct the error in DNA that causes beta-thalassemia. The team used a term called “base editing,” also known as chemical surgery, to alter the fundamental building blocks of DNA. Ultimately, correcting a single error out of the staggering three billion “letters” that make up our genetic code by converting one DNA base into another. The success of this procedure will hinge on the fact that life-threatening blood disorders stem from a change to just a single base in a person’s genetic code, known a point mutation.

The team at Sun Yat Sen University were able to successfully edit this single change and correct it. This work was done on lab-made embryos that were not implanted, however, the promise of this work cannot be overstated. David Liu, Professor of Chemistry and Chemical Biology at Harvard University, pioneered base editing, noted the importance of base editing. “About two-thirds of known human genetic variants associated with diseases that are point mutations. So base editing has the potential to directly correct, or reproduced for research purposes, many pathogenic [mutations].”

There are however a number of regulatory hurdles that will need to be surmounted. Jennifer Doudna, one of the world’s leading gene edit researchers noted, “I think it’s really likely that in the not-too-distant future it [gene editing] will cure genetic disease…but globally we need to come up with a consensus on moving forward in a responsible way.” The tool has an incredible potential, however, it will need to be carefully considered.

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