Congrats on this great work, @jmathony.bsky.social @dominikniopek.bsky.social & team!

Publication alert: Our paper on domain insertion predictions in proteins is now out in @natmethods.nature.com in its final form: rdcu.be/ey7w3
Also check out the nice perspective by @noahholzleitner.bsky.social and @grunewald.bsky.social : www.nature.com/articles/s41...

Our work on rationally engineering allosteric protein switches is now out in Nature Methods: www.nature.com/articles/s41... Thanks a lot to @grunewald.bsky.social and @noahholzleitner.bsky.social for the comprehensive news and views: www.nature.com/articles/s41...

Dual SORT LNPs for multi-organ base editing go.nature.com/3HC9EHt

For the first time, doctors have created a customized treatment using the revolutionary gene-editing technique known as CRISPR to treat a baby with a rare, life-threatening genetic disorder.

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...

Evolution-guided protein design of IscB for persistent epigenome editing in vivo - @zhangf.bsky.social @broadinstitute.org @hhmi.org go.nature.com/44XVBph

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

AlphaFold is amazing but gives you static structures 🧊

In a fantastic teamwork, @mcagiada.bsky.social and @emilthomasen.bsky.social developed AF2χ to generate conformational ensembles representing side-chain dynamics using AF2 💃

Code: github.com/KULL-Centre/...
Colab: github.com/matteo-cagia...

We are super excited to share our work on #InnateImmune sensing of #AAV 🦠🧬 in the 🧠, just out in @natcomms.nature.com ! 🤩

Huge congratulations to all the team and collaborators 💪🏻

And our funders 💰
@erc.europa.eu #FondazioneTelethon and #FNIH 🙏🏻

www.nature.com/articles/s41...

Our latest work now online in Cell:

Rewriting regulatory DNA to dissect and reprogram gene expression

Our new method (Variant-EFFECTS) uses high-throughput prime editing + flow sorting + sequencing to precisely measure effects of noncoding variants on gene expression

Thread 👇

RFDiffusion2: a diffusion model that scaffolds enzyme active sites at the atomic level enables the generation of functional de novo enzymes

www.biorxiv.org/content/10.1...

www.cell.com/cell/fulltex...
ENVLPE, our system for VLP-based delivery of gene editors, is now published in @cellcellpress.bsky.social. We showed efficient delivery of base- and prime-editors in primary T cells & in vivo retinal models. All core constructs are available from @addgene.bsky.social.

Dive into our lab’s first peer-reviewed publication—co-led with our friends @benadler.bsky.social and Muntathar Al-Shimary from the @doudna lab! If you’ve already read the preprint, don’t miss the new updates: doi.org/10.1038/s415... @naturemicrobiol.bsky.social

Making intrabodies from antibodies just got easier! Learn how we made 𝟭𝟵 intrabodies to bind and light up peptides and histone modifications in live cells. And thanks to Academia, all sequences are freely available. (video credit: Yuko Sato @YukoSatoT2) (1/15)
www.biorxiv.org/content/10.1...

Twitter:
LNP/mRNA therapeutics and AAV-based approaches require cell-level precision to achieve success without significant toxicity to other cells. To enable this, we developed SCP-Nano: an AI and tissue-clearing-based technology for precision nanotechnology. www.nature.com/articles/s41...

New paper from our lab (@erturklab.bsky.social)

We show how tissue clearing, light sheet microscopy, and AI-based image analysis can be combined to determine the biodistribution and drug delivery efficiency of nanocarriers throughout entire mouse bodies with single-cell resolution.

🧪1/ 🚨New paper on ecDNAs from our lab!
We reveal a strategy to engineer extrachromosomal DNA (ecDNA) amplifications in cells & mice. Let's dive in! Let’s dive in! 🧵👇
www.nature.com/articles/s41...

I could not be more thrilled to announce the Nature Methods @naturemethods.bsky.social Method of the Year is Spatial Proteomics! Please see our editorial as a roadmap to the fantastic content in this special issue! www.nature.com/articles/s41...

Imagine seeing a disease's impact on every cell in the body. Now, you can! We introduce MouseMapper, an AI tool to reveal system-wide disease perturbations. It reveals obesity-induced changes in facial nerves & body-wide inflammation 👉🧵https://biorxiv.org/content/10.1101/2024.08.18.608300v1.full.pdf

Our new preprint is out - please share!

We asked: How can we make CRISPR knockouts in vivo more efficient? And set up an assay that enables detection of CRISPR cutting with 100s-1000s of sgRNAs over entire chromosome arms in living animals.

Grab a ☕ and let’s dive in. 🧵🧪 1/n
#CRISPR #geneediting

By definition, enhancers can activate from a distance. But with increased distance between enhancer and promoter, the activation drops. To study this systematically, we build a synthetic locus: www.cell.com/molecular-ce... 1/12

2X #DilatedCardiomyopathy GWAS MATG #NatGenet 2024 reveal novel genes/cellular players

PROX1 - #LymphaticEndothelialCell
ERG - #EndothelialCell
DGKG - #SmoothMuscleCell
MAPT/Tau validated in both studies

>9k case
www.nature.com/articles/s41...

>14k case
www.nature.com/articles/s41...