Is gene therapy changing pediatric health care? | Boston Children’s Answers Parentcast (S1: Ep.12)

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:
1:05 What is gene therapy?
5:40 How does gene therapy work?
10:54 What are gene insertion and gene editing?
14:11 What are some common diseases treated by gene therapy?
18:51 How is gene therapy impacting rare diseases?
21:30 How common is gene therapy?
24:27 What causes genetic mutations?
27:49 Where can you find gene therapies for specific conditions?
29:45 What gene therapy options are offered at Boston Children’s Hospital?
34:05 Are there any risks with gene therapy?
40:05 Where can someone get gene therapy?
43:11 What might happen with gene therapy in the future?
46:46 What are the doctor’s orders regarding gene therapy?

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.

▶Meet the Experts:

Christine Duncan, MD
Medical Director of Clinical Research and Clinical Development, at Boston Children’s Gene Therapy Program
https://www.childrenshospital.org/directory/christine-duncan

Jennifer Arnold, MD, MSc
Neonatologist and Program Director, Immersive Design Systems
https://www.childrenshospital.org/directory/jennifer-arnold

▶Program mentioned:

Gene Therapy Program
https://www.childrenshospital.org/programs/gene-therapy-program

▶More Resources and Answers for Patients & Families:

FDA-approved gene therapies: https://www.childrenshospital.org/programs/gene-therapy-program/fda-approved-gene-therapies

A look inside Boston Children’s Gene Therapy Program: https://youtu.be/2qMUyIQ2sOE

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#genetherapy #podcast #kidshealth #bostonchildrenshospital #pediatrics #healthcare
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