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Today we report in Nature Biotechnology 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…

Excited to share our new system for evolving eVLPs with improved production and protein delivery efficiencies! Grateful to David R. Liu and liugroup for support throughout this study, and special thanks to co-authors Meirui An and Paul Chen for their contributions!

A new gene-editing treatment extends lifespan by about 50 percent in a mouse model of prion disease and could lead to a preventative, one-time treatment for the deadly neurodegenerative disorder. broad.io/Prion-GeneEdit…

Today we report in Nature Medicine the development of a base editing strategy for prion disease, currently a fatal and rapidly progressing neurogenerative condition with no effective treatment. @PrionAlliance DevermanLab (1/13) drive.google.com/file/d/1DVpKrz…

This year's Precision Medicine World Conference (Precision Medicine World Conference) is right around the corner! For those who can join, David R. Liu's presentation will be part of the "Cell & Gene Therapies" Track on 02/06. Full program lineup: pmwcintl.com/program/

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

Congratulations to HHMI Investigator David R. Liu (David R. Liu) on receiving the 2025 Breakthrough Prize (Breakthrough) in Life Sciences! He is recognized for developing base editing and prime editing: two breakthrough gene-editing technologies that enable the correction or

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 U.S. FDA for clinical trial use, manufactured as a complex with an LNP, and dosed into a baby with a severe,

Today in Science Magazine 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 Sternberg Lab. 1/13 drive.google.com/file/d/1I-UbCR…

Using laboratory-evolved versions of enzymes called CASTs, researchers have achieved a long-standing goal of genome editing: inserting entire healthy genes into human cells efficiently enough for potential therapeutic applications. broad.io/CASTs-news

A baby named KJ, who was born with a rare genetic disorder – CPS1 deficiency – is healed thanks to doctors at the Children's Hospital who administered the world’s first personalized gene-editing treatment. The research team used base editing, a gene-editing method invented by HHMI

Never could I have imagined that ABE would someday enable such a transformative outcome for a child. Thank you Kiran Musunuru, Rebecca Ahrens-Nicklas, and KJ’s family for having the courage to demonstrate to the world what is possible. Incredible. 💕 nytimes.com/2025/05/15/hea… via

A gene-editing treatment used on a 9½-month-old boy with a rare condition has the potential to help people with thousands of other uncommon genetic diseases #NBTNewsBeat nytimes.com/2025/05/15/hea…

Excited to be part of this work and be able to advance evoCAST to enable efficient and precise gene insertions into the human genome. Arguably the hardest project I've worked on, but also the most interesting and exciting. Thank you to the liugroup and Sternberg Lab!

“One of the biggest tragedies I've observed in my professional life.” David R. Liu, winner of the Breakthrough in Life Sciences and professor at the Broad Institute and Harvard University, describes the cost of government cuts to science research at universities. Walter Isaacson

I discuss the urgent crisis from the loss of federal support of science in the US and recent clinical gene editing breakthroughs with Walter Isaacson & Christiane Amanpour on Amanpour and Company, airing on PBS tonight at 11 pm ET, and on CNN International earlier today. Pls share! youtube.com/watch?v=8YhJM6…

In Nature Genetics we report that base editing of trinucleotide repeats (TNRs) reduces somatic repeat expansions in Huntington’s disease (HD) and Friedreich’s ataxia (FRDA)—in patient-derived cells and in vivo—a collaboration with the Mouro Pinto lab. drive.google.com/file/d/1on7kdy… 1/14

Researchers have developed a way to edit the genetic sequences at the root of Huntington’s disease and Friedreich’s ataxia. broad.io/GE-Huntingtons

The latest from Liu Group alum Luke Koblan? Oh, just a way to study cell state and lineage dynamics over biologically relevant spatiotemporal scales....in vivo 🧐🔬✨ Introducing PEtracer 👇🏼