How is gene therapy transforming pediatric health care?

Genes provide information/instructions that influence everything about you and your child, from the color of your eyes to the intricate functioning of your body. When a gene is missing or doesn’t work correctly, it can lead to various health issues. Enter gene therapy — a revolutionary technique that uses a person’s genes to treat or even cure diseases. In this episode, Dr. Jennifer Arnold talks with Dr. Christine Duncan, medical director of clinical research and clinical development, at Boston Children’s Gene Therapy Program. Together, they delve into the transformative impact of gene therapy on pediatric health care, especially in addressing rare diseases. Explore key questions with them, such as: • What are genetic mutations? • How does gene therapy work? • What makes gene therapy unique as a form of treatment? • Which pediatric diseases have FDA approved gene therapies today? • How does one learn if a gene therapy clinical trial exists for a specific condition? • Is gene therapy considered a safe approach? • What does the future of gene therapy look like? Get ready for a fascinating journey into the world of gene therapy and its profound impact on the future of health care — especially for kids.