People with sickle-cell disease, a group of inherited blood disorders, have abnormal hemoglobin in their red blood cells, causing blood to clog in the tiny vessels and organs of the body.
According to Dr. Marina Cavazzana, senior author of the study and head of the biotherapy department at Necker, “all the biological tests we perform lead us to think he is cured.” Yet, she added, the answer to the question of whether he is truly cured “can be provided only by the longer follow-up.”
Still, hopes are running high that patients with this very devastating disease can receive this therapy “in the next five years,” Cavazzana said. “This is our hope, and we work very hard to attain it.”
Worldwide, more than 275,000 infants are born with sickle cell disease each year. In the United States, approximately 100,000 people, most of African ancestry or identifying as black, currently have it. About one …
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