Two Gene Therapies For Sickle Cell Disease Approved In US
This electron microscope image provided by the National Institutes of Health in 2016 shows a blood cell altered by sickle cell disease, top. (National Center for Advancing Translational Sciences (NCATS), National Institutes of Health via AP)
(Associated Press) – U.S. regulators have approved two gene therapies for sickle cell disease.
Doctors hope they can cure the painful inherited blood disorder that afflicts mostly Black people in the U.S. One treatment is the first based on CRISPR, the gene editing tool that won its inventors the Nobel Prize in 2020.
The Food and Drug Administration approved the gene therapies on Friday.
While doctors say these therapies are promising, they point out there are still unknowns.
For example, they don’t know about possible long-term effects of the treatments.
