Gene Therapy: What Pediatricians need to know
Learning Objectives
By the end of this course, the learners should be able to:
- Describe the basic principles of genetics, gene delivery systems, and the major approaches used in gene therapy, including gene replacement, suppression, addition, and editing
- Identify current gene therapy technologies, such as viral vectors, antisense oligonucleotides, RNA interference, and CRISPR-based genome editing, and their clinical applications
- Recognize the role of gene therapy in the treatment of pediatric conditions including spinal muscular atrophy, Duchenne muscular dystrophy, inherited retinal diseases, sickle cell disease, beta-thalassemia, hemophilia, and pediatric leukemia
- Discuss the benefits, limitations, safety considerations, ethical challenges, and future directions of gene-based therapies in pediatric medicine
Intended Audience:
This online course is designed for pediatricians and family physicians.
Abstract:
This online course provides an overview of gene therapy and its rapidly expanding role in the treatment of pediatric diseases. Participants will explore the fundamental principles of genetics, gene delivery systems, and the major therapeutic approaches used in modern gene therapy, including gene replacement, gene suppression, gene addition, and gene editing. The course reviews current technologies such as viral vectors, antisense oligonucleotides, RNA interference, and CRISPR-based genome editing, highlighting how these innovations are transforming the management of previously incurable childhood disorders.
Through real-world clinical examples, including spinal muscular atrophy, Duchenne muscular dystrophy, inherited retinal diseases, sickle cell disease, beta-thalassemia, hemophilia, and CAR T-cell therapy for pediatric leukemia, participants will gain insight into the clinical impact, benefits, limitations, and future directions of gene-based therapies. The course also addresses safety considerations, ethical challenges, accessibility issues, and the evolving role of gene therapy in improving outcomes and quality of life for children with genetic diseases.
Assessment and Earning CME Credit:
This course in English is not CME-accredited. However, it provides valuable educational content that can enhance your knowledge and skills in the subject matter. To receive CME credit for this course in Armenia, you must take the course in Armenian and complete the post-course quiz. You have three chances to achieve a passing score of 70%. Once you pass the quiz, we will send your information to the Armenian Ministry of Health for credits.
Dr. Alan Wayne
Alan S. Wayne, MD, is Pediatrician-in-Chief and Senior Vice President of Academic Affairs at Children’s Hospital Los Angeles (CHLA), where he also serves as the Pasadena Guild Chair and Chair of the Department of Pediatrics. He is a Professor of Pediatrics and Medicine at the Keck School of Medicine of USC and oversees CHLA’s clinical, educational, and research initiatives, including The Saban Research Institute.
An internationally recognized leader in pediatric hematology-oncology and cellular therapies, Dr. Wayne’s clinical and research interests focus on hematologic malignancies, hematopoietic stem cell transplantation, cancer immunotherapy, and the development of targeted and gene-based therapies for children with cancer and other serious diseases. Prior to joining CHLA in 2013, he served for 14 years as Clinical Director of the Pediatric Oncology Branch at the National Cancer Institute. Dr. Wayne has received numerous honors for his contributions to pediatric medicine and continues to advance innovative therapies that improve outcomes for children worldwide.
