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04 - Drew Weissman, MD, PhD - Giants of Gene Therapy

ASGCT Podcast Network

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Advancements in mRNA and Gene Therapy

This chapter explores the breakthroughs in mRNA technology and lipid nanoparticles for treating diseases such as sickle cell disease, highlighting efforts in gene editing and potential clinical trials. It also discusses the challenges of public perception regarding RNA therapies and the need for effective delivery systems for broader access, especially in underdeveloped regions.

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