Advancing Gene Therapy: KJ’s Story

Advancing Gene Therapy: KJ’s Story

From BREAKING THROUGH with Madeline Bell by Children’s Hospital of Philadelphia

August 22, 2025 · 32 min · Episode 22

About this episode

This episode features KJ's parents discussing their child's groundbreaking gene therapy treatment at CHOP.

Earlier this year, baby KJ became the first patient in the world to receive a personalized gene editing therapy, which was developed by a team at Children’s Hospital of Philadelphia (CHOP) and Penn Medicine. In June, KJ went home after spending more than 300 days at CHOP. On this episode of Breaking Through, Madeline speaks to KJ’s parents, Kyle and Nicole, about the treatment that changed KJ’s life – and how he’s doing today.

People in this episode

Host: Madeline Bell

Guests: Kyle, Nicole

Topics covered

  • gene therapy
  • personalized medicine
  • pediatric health
  • patient stories
  • medical innovation

Keywords

  • gene editing
  • KJ
  • Children’s Hospital of Philadelphia
  • Penn Medicine
  • pediatric gene therapy
  • medical treatment
  • patient experience

Mentioned in this episode

Organizations: Children’s Hospital of Philadelphia, Penn Medicine

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