Gene editing pioneer David Liu unlocks new treatments for rare diseases

Liu's inventions like 'base editing' and 'prime editing' are transforming the field of genetic medicine.

Published on Feb. 9, 2026

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David Liu, a biochemist at the Broad Institute and Harvard University, has been at the forefront of a revolution in medicine, inventing powerful and precise tools to edit the code of life. His inventions like 'base editing' and 'prime editing' have opened up new possibilities for treating thousands of rare genetic diseases. Liu's work has already helped save the life of a baby named KJ, and he is now raising funds for a nonprofit Center for Genetic Surgery to expand access to these transformative treatments.

Why it matters

Rare genetic diseases affect over 400 million people globally, with half being children. Many of these patients have no treatment options, but Liu's gene editing breakthroughs offer hope for addressing the 'global health crisis hiding in plain sight'.

The details

Liu's lab has designed crucial components of personalized gene therapies, like the base editor used to treat baby KJ's life-threatening metabolic disease. His team has also created an even more versatile editor called 'prime editing' that has successfully treated genetic mutations in two patients. Liu has co-founded multiple gene-editing companies and is now raising funds for a nonprofit Center for Genetic Surgery at the Broad Institute to focus on rare diseases that lack commercial viability for biotech and pharmaceutical companies.

  • In 2016, Liu's invention of 'base editing' opened the possibility of fixing certain genetic errors.
  • In 2022, Liu's 'prime editing' technology was successfully used to treat genetic mutations in two patients.
  • In June 2025, after 307 days in the hospital, baby KJ went home after receiving the personalized gene therapy.

The players

David Liu

A biochemist at the Broad Institute and Harvard University who has been a pioneer in developing powerful gene editing tools like 'base editing' and 'prime editing' to treat rare genetic diseases.

KJ

A baby who received a personalized gene therapy developed with Liu's 'base editing' technology to treat a life-threatening metabolic disease.

Waseem Qasim

A professor of cell and gene therapy at University College London-Great Ormond Street Institute of Child Health who began using Liu's base editing technology for immune cell treatments in children with a form of childhood blood cancer.

Alyssa Tapley

A 13-year-old patient who received a gene therapy treatment using Liu's base editing technology, allowing her to say 'I wasn't ready to die. I hadn't done anything yet.'

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What they’re saying

“This is impressive. And I wonder if it is true. The chemistry was so clever.”

— Waseem Qasim, Professor of cell and gene therapy (N/A)

“I wasn't ready to die. I hadn't done anything yet. I'm 16, preparing for my exams, spending time with my family, arguing with my brother, and doing all the things I thought that I would never be able to do. But most importantly, I have a future.”

— Alyssa Tapley (N/A)

What’s next

Liu is raising funds for a nonprofit Center for Genetic Surgery at the Broad Institute to expand access to these transformative gene editing treatments for rare diseases that lack commercial viability.

The takeaway

David Liu's pioneering work in gene editing has the potential to transform the lives of millions of people suffering from rare genetic diseases, offering hope where there was previously little to none. His inventions are already saving lives and paving the way for a new era of personalized genetic medicine.