Paul Chen

Today in @Nature we report a new prime editing strategy that can rescue a common cause of many genetic diseases in a disease-agnostic manner. This approach converts a redundant endogenous tRNA into an optimized suppressor tRNA, enabling a single prime edit to rescue premature stop codons across different diseases. (1/15) drive.google.com/file/d/1bSvkJW…

Science delivers medicines, clean air and water, safe food, and smarter choices—saving billions of lives. Proposed cuts to @NIH and @NSF would shutter labs, stall new cures, hurt our economy, and send future scientists elsewhere. Hear from patients saved by science: tinyurl.com/m65339fh. To share your story of how #ScienceSavedMe, email sciencesavedme@gmail.com. 1/2

A wonderful article by @DhruvKhullar on patients with progeria, the genetics behind this rare disease, and our ongoing efforts with PRF and @NIH to advance promising base editing outcomes in humanized progeria mice into a clinical trial. newyorker.com/magazine/2025/…

Today in @CellCellPress we report the use of prime editing to correct several mutations that cause alternating hemiplegia of childhood (AHC), a rare and devastating neurodevelopmental disorder, in patient-derived cells and in two mouse models. drive.google.com/file/d/1ibC50t… 1/10

Absolutely thrilled to make public that I am joining the @harvardmed Harvard Medical School and MGB @CGM_MGH Center for Genomic Medicine and @MGHNeurology Department of Neurology as an Assistant Professor: stantonlab.info. 🧵

I discuss the urgent crisis from the loss of federal support of science in the US and recent clinical gene editing breakthroughs with @WalterIsaacson & @amanpour on @AmanpourCoPBS, airing on @PBS tonight at 11 pm ET, and on @cnni earlier today. Pls share! youtube.com/watch?v=8YhJM6…

Another medical milestone! The first patient treated with a prime edited therapeutic shows positive clinical outcomes consistent with effective rescue of his chronic granulomatous disease (CGD), a debilitating and life-shortening primary immunodeficiency. At least 19 base editing and prime editing clinical trials have begun, with at least 7 trials reporting clinical outcomes (highlighted in green in the table below). They treat a wide range of diseases both in vivo and ex vivo. And while the U.S. support for science is now in crisis, that there are more base editing and prime editing clinical trials serving patients outside of the U.S. (13) than within the U.S. (9), despite the fact that these technologies were developed in our lab in the U.S. investors.primemedicine.com/news-releases/…

1/10 Today in @ScienceMagazine in collaboration with @liugroup we report the development of a laboratory-evolved CRISPR-associated transposase (evoCAST) that supports therapeutically relevant levels of RNA-programmable gene insertion in human cells. drive.google.com/file/d/1I-UbCR…

A baby named KJ, who was born with a rare genetic disorder – CPS1 deficiency – is healed thanks to doctors at the @ChildrensPhila who administered the world’s first personalized gene-editing treatment. The research team used base editing, a gene-editing method invented by HHMI Investigator @davidrliu and his lab at @Harvard University in 2016. Earlier this year, Liu received the @brkthroughprize for developing both base editing and prime editing. These gene-editing technologies enable the correction or replacement of virtually any genetic mutation, including those that cause countless human genetic diseases. Read more about Liu's work here: hhmi.org/news/david-liu…

CHOP & @PennMedicine have delivered the first-ever personalized gene editing therapy for a patient with CPS1 deficiency, marking a major milestone in the application of CRISPR-based treatments. Learn more about KJ & the future of personalized medicine: ms.spr.ly/6015SZLAx.

Today in @ScienceMagazine we report the development of a laboratory-evolved CRISPR-associated transposase (evoCAST) that supports therapeutically relevant levels of RNA-programmable gene insertion in human cells, a collaboration with @SternbergLab. 1/13 drive.google.com/file/d/1I-UbCR…

In a medical milestone, a customized base editor was developed, characterized in human and mouse cells, tested in mice, studied for safety in non-human primates, cleared by @US_FDA for clinical trial use, manufactured as a complex with an LNP, and dosed into a baby with a severe, rapidly progressing genetic disease... all in an astounding 7 months. Best of all, the infant patient shows apparent benefit. Congratulations to @kiranmusunuru, Rebecca Ahrens-Nicklas, and other team members for this heroic and inspiring effort, which has implications for the hundreds of millions of patients that suffer from thousands of genetic diseases. drive.google.com/file/d/1Jfku5j…

Imagine a world without MIT.

I'm excited to share that I will be joining @UclaCBE as an Assistant Professor starting July 2027!

Last night I was honored to receive the 2025 #BreakthroughPrize in the Life Sciences, reflecting the efforts of many students, collaborators, doctors, and patients in labs around the world. I hope this 4-minute excerpt can inspire when science needs public support more than ever.

David Liu wins the Breakthrough Prize in Life Sciences for developing gene-editing technologies, including a "molecular word processor" that can find and replace whole sequences of DNA. His tools are already impacting diseases including cancer. breakthroughprize.org/News/91 @broadinstitute @HHMINEWS @Harvard

David Liu receives Breakthrough Prize in Life Sciences. @davidrliu is honored for the development of base editing and prime editing, two gene editing technologies transforming medicine. @brkthroughprize broadinstitute.org/news/david-liu…

Privileged to share initial data for BEAM-302 today in alpha-1 antitrypsin deficiency (AATD)! AATD is a severe, progressive disease impacting both lung and liver function that affects 100,000 people in the US globenewswire.com/news-release/2…

Excited to share our new system for evolving eVLPs with improved production and protein delivery efficiencies! Grateful to @davidrliu and @liugroup for support throughout this study, and special thanks to co-authors Meirui An and @paulchenz for their contributions!

Today we report in @NatureBiotech a system for the laboratory evolution of engineered virus-like particles (eVLPs) that enables the discovery of eVLP variants with desired properties, including improved production and delivery potency. 1/13 PDF: drive.google.com/file/d/1E6Nr86…