“Sickle cell, an inherited blood disorder, causes red blood cells to become misshapen half-moons that get stuck inside blood vessels, restricting blood flow and causing what are known as pain crises. About 100,000 Americans are estimated to have the disease.
Casgevy uses CRISPR to make an edit to a person’s DNA that turns on fetal hemoglobin, a protein that normally shuts off shortly after birth, to help red blood cells keep their healthy full-moon shape. In clinical trials, Casgevy eliminated pain crises in most patients.
The FDA approved the treatment for people 12 years and older.”
From NBC Yew York.