
NEJM This Week — June 12, 2025
NEJM This Week
00:00
Advancements and Challenges in Gene Editing for Rare Diseases
This chapter explores the complexities of personalized treatments for rare inborn errors of metabolism, stressing the need for thorough evaluation of gene editing therapies. It highlights the significance of longitudinal studies and addresses ethical challenges in moving from initial proof of concept to standard healthcare practices.
Transcript
Play full episode