Tag: CRISPR

Sickle cell therapy pioneered at CHOP a "real gamechanger" Sickle cell therapy pioneered at CHOP a "real gamechanger" 02:40 PHILADELPHIA (CBS) – A new medical breakthrough has roots in Philadelphia. The Food and Drug Administration recently approved the first gene therapies to treat patients with sickle cell disease, an inherited blood disorder that strikes mainly people of color. "You feel like somebody is taking a sledgehammer to your bones and hitting it over and over again," said...

PHILADELPHIA (CBS) – A new medical breakthrough has roots in Philadelphia. The Food and Drug Administration recently approved the first gene therapies to treat patients with sickle cell disease, an inherited blood disorder that strikes mainly people of color. "You feel like somebody is taking a sledgehammer to your bones and hitting it over and over again," said Daniels Tornyenu, who has sickle cell disease. His daughter, Marie-Chantal Tornyenu, has the illness as well. "In my chest, it's like this hollow burning almost, and then I can't breathe on top of it," Marie-Chantal Tornyenu said. RELATED: FDA approves gene-editing treatment for sickle cell diseasePain from sickle cell can happen anywhere blood circulates. Because red blood cells, which are normally round, bend into inflexible s...