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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.
By Children’s Hospital of Philadelphia4.7
1616 ratings
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.

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