When baby KJ was diagnosed with a rare metabolic disorder, the team at Children’s Hospital of Philadelphia (CHOP) had a unique treatment option for him: a personalized gene editing therapy developed in collaboration with Penn Medicine. On this episode, Madeline speaks to CHOP geneticist Dr. Rebecca Ahrens-Nicklas, who describes how KJ’s first-of-its-kind treatment was developed – and what this amazing breakthrough means for other patients. 

BREAKING THROUGH with Madeline Bell

Children’s Hospital of Philadelphia

Advancing Gene Therapy: Personalized Gene Editing

OCT 13, 202522 MIN
BREAKING THROUGH with Madeline Bell

Advancing Gene Therapy: Personalized Gene Editing

OCT 13, 202522 MIN

Description

When baby KJ was diagnosed with a rare metabolic disorder, the team at Children’s Hospital of Philadelphia (CHOP) had a unique treatment option for him: a personalized gene editing therapy developed in collaboration with Penn Medicine. On this episode, Madeline speaks to CHOP geneticist Dr. Rebecca Ahrens-Nicklas, who describes how KJ’s first-of-its-kind treatment was developed – and what this amazing breakthrough means for other patients.