liugroup

Just a reminder that *all* of our lab's published papers can be freely accessed at any time on our lab webpage here: liugroup.us/publications #openscience #genomeediting #geneediting #chemtwitter

Another jump for genome editing today by @davidrliu and colleagues, addressing the ~11% of nonsense mutations that lead to premature stop codons, affect protein synthesis, and suitable for disease agnostic intervention @Nature @NatureNV nature.com/articles/s4158… nature.com/articles/d4158…

A team at the Broad Institute co-led by @Sarah_E_Pierce, @stevenerwood, and @davidrliu has developed a new genome-editing strategy that could potentially lead to a one-time treatment for multiple unrelated genetic diseases. broad.io/PERTNews

Single Prime Editing Platform Shows Potential vs. Multiple Diseases PERT edits the genome to permanently express a suppressor #tRNA, so that cells affected by nonsense mutations can produce functional protein @davidrliu #primeediting #geneticdiseases hubs.li/Q03Vdkqc0

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…

That @US_FDA is recognizing here the urgent need for regulatory strategies to evolve to match the pace of scientific advances in ways that streamline bringing the science safely to patients is incredibly important. I hope these new policies provide a foundation that will eventually allow certain kinds of genetic surgeries to become as routine as life-saving conventional surgeries, in which the process is robust and standardized enough to be rapidly adapted to each patient, without the need for a separate multi-year regulatory process for each surgery.

📣 Announcing the 2026 Franklin Institute Awards Laureates! Their work spans a range of scientific discoveries, from the smallest units of life to the vast expanse of the universe. Visit fi.edu/awards to learn how they are charting bold paths to the future! #FIAwards

Alyssa Tapley was 13 when doctors told her she was going to die. She was diagnosed with a rare form of leukemia and had endured a year of failed treatments, including chemotherapy and a bone marrow transplant, when her doctors said they had run out of options. They were talking about end-of-life care when Tapley begged them not to give up on her. cbsn.ws/4r5E0EO

Tonight’s episode of @60Minutes will include a segment on the impact of federally funded science and recent cuts to science funding. Our lab’s development of base editing and prime editing and their clinical applications will be among the work described in the episode. paramountpressexpress.com/cbs-news-and-s…

#NASmember David Liu is a pioneer in developing techniques to treat genetic disorders, including CRISPR. In an interview with @sciencenews, he discusses what first drew him to science, and what gives him hope for its future. Read here: ow.ly/w9BH50XcPOj #genetics

An excellent interview with @davidrliu in Harvard Magazine highlighting his lab’s inventions (PACE, base editing, prime editing), his high-energy work ethic and “ceaseless” drive, his mentoring philosophy (giving freedom, emphasizing collaboration), and his motivation to translate basic research into patient impact. harvardmagazine.com/science/harvar…

“Taking advantage of the ability of prime editing to do search-and-replace gene editing raises the possibility that a single composition of matter [...] might benefit many, many more patients than going after diseases one mutation at a time” -@davidrliu wired.com/story/the-next…

When I first joined @GENbio in 2022, there was one name I kept hearing seemingly weekly: @davidrliu. David Liu's lab invented base and prime editing (gene editing tools now in 20+ clinical trials with early wins against sickle cell, leukemia, and more). In this episode of @Inside_PM's Behind The Breakthroughs, David shares about his childhood, how he landed on the problem of engineering gene editors, and some of what he plans to reveal to the world next. Liu is a professor at @Harvard University and the @broadinstitute, co-founder of several companies, including @editasmed, @BeamTx, and @PrimeMedicine, and winner of the 2025 Life Sciences award from the @brkthroughprize. shows.acast.com/67105af8487269… via @acast

One-shot prime genome editing ("CRISPR 3.0") of the brain in vivo corrected AHC, a rare genetic disorder, in the mouse model. Another big step forward in the genome editing field. @CellCellPress @davidrliu and colleagues cell.com/cell/fulltext/…

These results represent the first use of prime editing to rescue a neurological disease in animals, establish that in vivo postnatal intervention in AHC’s pathology is possible, and suggest the potential of a one-time prime editing treatment for AHC. (Photo: hopeforannabel.org/ahc/) 9/10

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

Partnership between @davidrliu, @jacksonlab, and rare disease group @RAREH0pe leads to first use of prime editing for a brain disease that reduced symptoms, extended lifespan in mice, offering hope for other rare genetic diseases. broad.io/Prime-AHC

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 👇🏼

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

In @NatureGenet 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

“One of the biggest tragedies I've observed in my professional life.” @davidrliu, winner of the @brkthroughprize in Life Sciences and professor at the @broadinstitute and @Harvard, describes the cost of government cuts to science research at universities. @WalterIsaacson