Patient May Become First Person in Louis

Louisiana, is hoping to make history as the first person in the state to be cured of sickle cell disease using a groundbreaking new gene therapy.
Daniel, who dreams of one day becoming a pilot, has been told he can only obtain a pilot’s license if he is fully cured of the disease.

On Wednesday, he began the first step toward that goal at Manning Family Children’s Hospital, where he underwent the first of two six-hour sessions to collect his stem cells.

According to WWLTV, those cells will be sent overseas for genetic editing, designed to correct the defect that causes his body to produce misshapen,

sickle-shaped red blood cells.

The edited cells will then be reintroduced into his body, with the goal of allowing him to produce healthy, normally shaped red blood cells.

“Honestly, it’s a relief,” said Daniel. “It’s been a long time coming, and this is such a big step. We’re just hoping they collect enough cells so the process can move forward quickly.”

Until now, the only long-term treatment for sickle cell disease has been a bone marrow transplant, which requires a close genetic match—something not all patients have. This new therapy offers an alternative for many who have had few options.