Broad Institute
@broadinstitute
A multidisciplinary community of researchers on a mission to improve human health.
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/…
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

.@Schmidt_Center fellows Divya Shyamal & @JiaqiZhangVic, with senior author Caroline Uhler, introduce a new framework for designing experiments with treatment combos, scaling discovery of complex interactions in biology and medicine. 🔗 news.mit.edu/2025/more-effi… @broadinstitute
Join me for this lecture to find out how we use genomics to decipher #autoimmunity and #rheumatoid arthritis
Join us on Thursday, August 14 at 6 PM ET in Cambridge, MA – or online – for a Broad Discovery Series talk with @soumya_boston: “Arthritis, autoimmunity, and how the genome works.” Register: broadinstitute.swoogo.com/DiscoverySerie… #ScienceTalks
I've always found @soumya_boston's research to be absolutely fascinating, and I'm excited to bring him on-stage for the next talk in the @broadinstitute's Broad Discovery Series. Join us in-person or online on 8/14!
Join us on Thursday, August 14 at 6 PM ET in Cambridge, MA – or online – for a Broad Discovery Series talk with @soumya_boston: “Arthritis, autoimmunity, and how the genome works.” Register: broadinstitute.swoogo.com/DiscoverySerie… #ScienceTalks
Great opportunity to hear from our very own @BrighamWomens researcher @soumya_boston!
Join us on Thursday, August 14 at 6 PM ET in Cambridge, MA – or online – for a Broad Discovery Series talk with @soumya_boston: “Arthritis, autoimmunity, and how the genome works.” Register: broadinstitute.swoogo.com/DiscoverySerie… #ScienceTalks
Delighted to have our preprint led by @lvchosen1 up: Genetic variation reveals a homeotic long noncoding RNA that modulates human hematopoietic stem cells biorxiv.org/content/10.110… 🧵 below ...
We are thrilled to announce that Seth Klarman will become the next Chair of the Broad Institute Board of Directors, succeeding Eric Schmidt. We also welcome Aviv Regev as a new member of the Board. broad.io/Klarman-Board

Join us on Thursday, August 14 at 6 PM ET in Cambridge, MA – or online – for a Broad Discovery Series talk with @soumya_boston: “Arthritis, autoimmunity, and how the genome works.” Register: broadinstitute.swoogo.com/DiscoverySerie… #ScienceTalks

[1/10] At last, @MassGenBrigham x @MountSinaiNYC x @PennMedicine x @VUMChealth collaboration on external validation of the @AHAScience's PREVENT is now out in @JACCJournals! sciencedirect.com/science/articl…
An absolute risk generalizable risk calculator is challenging. Led by @somijemmacho, evaluating AHA PREVENT across 4 academic health systems, we found similar discrimination but variable calibration. A universal absolute risk threshold may not suitably guide.…
The July issue is live nature.com/nbt/volumes/43… On the cover, Chen et al. have developed a precise chemo-enzymatic method to synthesize and modify protein-coding mRNA to optimize functionality and pharmacokinetics go.nature.com/47EXbfq
Our paper on establishing targeted therapy for patients with myeloma and 1q+ is out in @BloodPortfolio! w/ Liz Lightbody, Mairead Reidy, @gaddyg @IrenemGhobrial and Salomon Manier @DanaFarber @broadinstitute #mmsm ashpublications.org/blood/article-… 1/
Our study led by R. Bhattacharya & @cmarnell92 shows that, when simultaneously considering clinical, laboratory, and genetic risk factors for incident CAD prediction, the top 2 model explanatory factors are: 1. Hypertension 2. CAD polygenic risk score ahajournals.org/doi/10.1161/CI……
Delighted to share our latest, expertly led by @KatlynGabriel_ and @ChoahK: FAF2 is a bifunctional regulator of peroxisomal homeostasis and saturated lipid responses Impactful work with therapeutic potential by our @GrekaLab team at @broadinstitute! science.org/doi/10.1126/sc…
Congratulations to @MagdalenaRiedlK on receiving the 2025 Ben J. Lipps Research Fellowship award from @ASNKidney!
👏 Congratulations to the 23 KidneyCure grant recipients for 2025! This group of emerging investigators join a cohort of 22 continuing grantees from the most prestigious research institutions in the United States. Meet the recipients: asn.click/45z5nPd
⚡🦠I'm excited to share that our latest work on the gut microbiome and metabolic dysfunction-associated steatotic liver disease (MASLD) is now published in @NatMetabolism, with @hutlab & @LongNguyen07! Check out the paper here: 🔗nature.com/articles/s4225… 👇Tweetorial
“Prof. Xiao Wang’s caring efforts have a profound impact on the lives of her students.” As part of the Committee to Caring that aims to develop extraordinary researchers and contribute to a collaborative culture, Wang excels in supporting student growth. chemistry.mit.edu/chemistry-news…
Congratulations to Broadies @zhangf and David Pellman on being elected as associate members of EMBO! embo.org/press-releases…