CRISPR


Kiran Musunuru Has Good News about Gene Therapy for Common Diseases

A cardiologist and Professor of Medicine at the University of Pennsylvania Perelman School of Medicine, Kiran Musunuru is a clinician and a researcher whose important work has moved the ball forward on gene therapy. As co-founder and scientific advisor to Verve Therapeutics, Kiran has a special perspective – an insider’s view of the business, from someone who is both an academic (MD, PhD, MPH) and a humanist at heart. No one better to discuss the news that broke last month, with early good reports on treatment for ATTR amyloidosis that uses lipid nanoparticles to deliver a CRISPR package for in vivo editing. Is this the breakthrough moment we’ve been waiting for since the Human Genome Project began? Yes, says Kiran Musunuru, going all-in on the next 5 years.

Walter Isaacson, "The Code Breaker"

Walter Isaacson, who has written biographies that explore the birth of the atomic age (Einstein) and the digital age (Jobs), now turns his attention to the biotech revolution, as embodied by CRISPR pioneer Jennifer Doudna with his new book, The Code Breaker. The discovery of CRISPR and its transformation from a bacterial system of immunity to a biotech toolkit was not a one-woman show, and I wondered if this famous chronicler of visionaries might take sides in what has become an occasionally less-than-genteel battle for credit, prizes, and patents.  But NO – this book opens up to cover a broad cast of characters, each of them given their due – so that Jennifer Doudna’s story is not a mere lionization but an entry point to a bigger, broader tapestry, the first draft of one of the most important moments in the history of science.

 

 

 

Hank Greely: CRISPR People

I can hardly believe I’ve been doing this podcast for more than 2 years and never had Hank Greely on before. Hank Greely is the Deane F. and Kate Edelman Johnson Professor of Law at Stanford University, and Director of the Center for Law and the Biosciences. When you want an opinion on anything at the intersection of law, policy, and bioethics, Hank is where you go. And I want an opinion on everything at the intersection of law, policy, and bioethics. Luckily, Hank’s new book, CRISPR People, brings him on the podcast today.

Kiran Musunuru, Verve Therapeutics

Kiran Musunuru, Associate Professor at the University of Pennsylvania Perelman School of Medicine, is back on The Beagle to talk about Verve Therapeutics, a company that announced its arrival earlier this month, with the modest goal of protecting the world from heart disease (honestly, “protect the world form heart disease" IS the Verve tagline).

Verve’s ambitions are founded upon work by Kiran, among others, that is directed at using gene therapy to fix – or even prevent – high cholesterol levels associated with increased risk of heart disease, the single most likely cause of death in adults. A small number of people carry a naturally-occurring variant that keeps their cholesterol levels low throughout their lives.

Can we use a CRISPR-based therapy to mimic that effect in people with sky-high lipid levels? And what about the rest of us? Kiran and the co-founders of Verve ask us to imagine a world where gene therapy is used like a vaccine.



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