Core Memory The State Of Gene Editing - EP 37 Eryney Marrogi
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Oct 8, 2025 Join Eryney Marrogi, a dedicated scientist and future medical doctor, as he dives deep into the exciting world of gene editing. He shares the remarkable story of Baby KJ, who received rapid, life-saving gene therapy, and discusses the implications for future treatments. Marrogi also explores the challenges of DIY bio, the alarming youth vaping trend, and why gene therapies are still costly. Plus, he reveals his transition from research to medicine, aiming to bring cutting-edge science directly to patient care.
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How Baby KJ’s Case Unfolded
- KJ was born with CPS1 deficiency causing toxic ammonia buildup and severe early metabolic crises.
- Doctors at CHOP recognized the mutation quickly and mobilized a personalized gene-editing plan built from prior drills.
A Simple Framework For Designing Gene Therapies
- Gene therapy design breaks into three questions: what organ to target, what gene to fix, and how long the fix should last.
- Those choices determine payload (editor), guide RNA, and delivery vehicle selection for a therapy.
Editors And Delivery Are The Core Bottlenecks
- Editing tools include CRISPR-Cas9, base editors and prime editors, each suited to different edit types.
- Delivery options (LNPs, viral vectors) control which organs you can reach and shape feasibility.