An inspirational panel this morning with none other than baby KJ himself - highlighting the stories of heroes @ahrensnicklas.bsky.social & @kiranmusunuru.bsky.social ( hosted by @jasonmast.bsky.social). Crescendo: KJ's Dad on watching 🏈 with his son: "This is what we thought we would never get". ❤️👏

Congrats to @connorjtou.bsky.social on being named a 2025 #STATWunderkind. His enthusiasm in the lab is contagious and he thrives on doing really hard things. Really well deserved recognition for an innovator and emerging leader in our field! 👏 🙌 @mgbresearch.bsky.social @harvardmed.bsky.social

Back in June, at our MSMDS conference, we shared how researchers were advancing their work on gene editing for ACTA2 R179H. Today, we can celebrate that this effort is now peer-reviewed & published in @nature.com! 🎉 t.co/6WjTMRnAOi #MSMDS #ACTA2 @markelindsay.bsky.social @bkleinstiver.bsky.social

Optimization of a bespoke base editor to treat a severe pediatric vascular disease! 🫀🧬 Our manuscript describes: 1️⃣ Engineering a target-specific BE🧬 2⃣ A *must avoid* bystander edit that occurs with WT SpCas9 BEs! 🙅‍♂️ 3⃣ Extension of lifespan after in vivo editing! 🐁✅ www.nature.com/articles/s41...

Exciting to see our protein binder design pipeline BindCraft published in its final form in @Nature ! This has been an amazing collaborative effort with Lennart, Christian, @sokrypton.org, Bruno and many other amazing lab members and collaborators. www.nature.com/articles/s41...

If you like transposons... If you you love genome editing... Or if you just like random bird animations, we have the paper for you! We (@kedmonds.bsky.social et al) are happy to share our work turning a songbird retrotransposon into a genome editing tool. 🐣 (1/n)

🚨 Our work on optimizing single-stranded DNA donors with Cas12a binding moieties is published at @moltherapy.bsky.social Nucleic Acids! 🧪 Since the preprint, we added two super interesting findings. Short 🧵👇 (1/5) www.cell.com/molecular-th... #ImmunoSky #GeneEditing #CARTcells

Less than one day to celebrate a major win for science, before returning to our regularly scheduled programming of destruction of the American scientific enterprise and capitulation..

1/10 Today in @science.org in collaboration with the Liu group 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-Ub...

Genomes encode biological complexity, which is determined by combinations of DNA mutations across millions of bases In new work @arcinstitute.org, we report the discovery and engineering of the first programmable DNA recombinases capable of megabase-scale human genome rearrangement

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 www.nytimes.com/2025/05/15/h...

An incredible collaborative effort led by @kiranmusunuru.bsky.social @ahrensnicklas.bsky.social @urnov.bsky.social & others. Congratulations to @rachelsilverstein9.bsky.social who designed the Cas9 enzyme that went into this drug. Wishing K.J. & his family all the best 🙏

Today "a milestone in the evolution of personalized therapies for rare & ultra-rare inborn errors of metabolism" —the 1st human to undergo custom genome editing —from decades of NIH funded research www.nejm.org/doi/full/10.... @nejm.org www.nejm.org/doi/full/10.... www.nytimes.com/2025/05/15/h...

Yesterday, the NIH R35 “Outstanding Investigator” grant to fund scientists in my lab studying antibiotic resistance was terminated for reasons not related to the content of the science, or any actions taken by me or members of my lab

pCMV-T7-SpCas9(MKRCMV)-P2A-EGFP (AHK162) Depositor: Benjamin Kleinstiver @bkleinstiver.bsky.social Purpose: pCMV and pT7 Human expression plasmid for SpCas9 enzyme with MKRCMV amino acid substitutions www.addgene.org/223075/

The genome-wide landscape of Cas-induced double-strand breaks and end structures is profiled at nucleotide resolution go.nature.com/44DO2Cc rdcu.be/ekKtv

By combining high-throughput protein engineering with machine learning, bespoke SpCas9 enzymes are identified that outperform evolution-based and engineered nucleases and base editors in human cells. www.nature.com/articles/s41... #NBTHighlight

In @nature.com we describe the use of scalable #proteinengineering & #machinelearning to predict millions of bespoke CRISPR enzymes, offering safer & more efficient genome editing tools 🧬🖥️ @rachelsilverstein9.bsky.social @mgbresearch.bsky.social @harvardmed.bsky.social www.nature.com/articles/s41...

Cas9 gets an upgrade! Check out our new paper in The CRISPR Journal: bit.ly/hiNLS-Cas9 Eric Noel (who is on the job market!) created Cas9 constructs w/ nuclear localization signals (NLS) in the backbone, boosting genome editing activity in T cells, as seen by editor @srishtisahu.bsky.social #CRISPR

Interested in learning more about genome editing tool dev & their application to treat human diseases? 🧬 🏥 We're hiring graduating seniors who are seeking ~2 years of post-bac research experience. If you're interested in gaining experience in next gen medicines, please reach out and apply! Link 👇

Bluetorial: Women, courage, and leadership What follows will include some generalizations based on population averages of what I have experienced over the course of my career. There are, of course, exceptions in every group who are substantially more to one extreme or the other.

If you're unsure whether advocacy or protest to save life-saving research matters, an important message here starting at 33:00. Together we can prevent devastating healthcare & economic impacts, and save science that doesn't see red or blue.. We need your support! ✊🧬🔬🧑‍🔬 youtu.be/cw0F8G4-dMw?...

Have you been interested combining Perturb-Seq screening with Cas12a’s unique capabilities for multiplexed guide RNA expression? Us too! So, we figured out some useful tricks to make that possible, which you can find in our new pre-print. Short thread below: www.biorxiv.org/content/10.1...

Very excited to share our new pre-print! We’ve developed a new way to put any type of cargo (biological or synthetic) into protein cages! All in-vitro! This enables massively scalable in vitro packaging technology for multiple lucrative applications. www.biorxiv.org/content/10.1...

Very excited about the first preprint from our lab at TUM! This is a fun gene & cell therapy collaboration led by @schmidts.bsky.social's lab. We use generative #AI to design new #CARTcell therapies. Check out Andrea's thread & thanks to the whole team! www.biorxiv.org/content/10.1...

Cancer escapes immunotherapies by mutating its surface, meaning even groundbreaking #CARTCell therapies fail too often. But what if #GenerativeAI could design CAR-T with de novo binders faster, targeting resistant tumors? 🧵 www.biorxiv.org/content/10.1... (1)

I took biochem in 2001, and for nearly 20 years read amino acid sequences daily… and I never knew Dayhoff named them or even the logic behind things like Q until last Friday (h/t Mike Janech). Also, this is another big Dayhoff moment for me. She was incredible! #proteomics #bioinformatics

We are pleased to share a preprint on the gene curation progress from the first 4 years of the ClinGen Syndromic Disorders Gene Curation Expert Panel (GCEP).

new building, same problems

Happy Friday! New preprint to discuss, led by @ekaplan.bsky.social, entitled "Activity-based selection for enhanced base editor mutational scanning" www.biorxiv.org/content/10.1... A thread...