CHOC Pediatrica
News and Information for Pediatric Healthcare Professionals
The 2025 administration of gene therapy to treat sickle cell disease marked more than an historic first for Rady Children’s Health Orange County. It also changed the trajectory of a young adult’s life and capped a two-decade journey for the physician and the hematology team who delivered it.
This year, more than 1,140 students applied for 75 internship positions, a reflection of both the program’s reputation and the growing interest among young people who want early exposure to healthcare.
Through a combination of formal instruction, mentorship, shadowing, and active participation in research activities, the CRC interns gained practical skills that cannot be fully taught in a classroom setting or abbreviated program.
Learn how Rady Children’s Health Orange County investigates gene therapy to treat childhood diseases, including Hurler syndrome, liver cancer and more.
‘I want families to know they have a trusted partner when an injury happens. I believe in taking time to educate patients and parents so they understand both the diagnosis and the recovery process.’
A third patient is scheduled to receive the FDA-approved therapy, with some patients showing improvement in their function and motor skills.
