New 🧬✂️ pre-print! We show that paired prime editing can efficiently generate large deletions — even >1 Mb — with high precision and at scale. We use this to perform the first pooled prime deletion screen across the human genome. 🔗 biorxiv.org/content/10.1... A short thread (by Juliane Weller)👇

The SCGE platform now has the first sets of researchers' regulatory documents available to the public. These documents are from regulatory interactions that SCGE researchers have had with the U.S. Food and Drug Administration (FDA). Access these documents at: scge.mcw.edu/platform/pub...

Read more about today's paper (tinyurl.com/23c5vzd9) from @ahrensnicklas.bsky.social & @kiranmusunuru.bsky.social that discusses regulatory innovation in the pathway toward interventional genetics!

Can personalized treatment become the standard of care? Just published in @ajhgnews.bsky.social, @kiranmusunuru.bsky.social, MD, PhD, and @ahrensnicklas.bsky.social, MD, PhD, explore their vision for the future of interventional genetics: www.cell.com/ajhg/fulltex... #ASHG #HumanGenetics

Super cool and important new work in gene dysregulation by HMGN1 as a mechanism for heart defects #CHD in Down Syndrome. From my @gladstoneinst.bsky.social colleagues led by @sanjeevranade.bsky.social www.nature.com/articles/s41...

The astonishing science of synthetic embryology and our profile of it's most ambitious praticioner, Jacob Hanna www.technologyreview.com/2025/10/21/1...

“We must keep going with this ability to sample genomes, not just in the convenient places we started out with.” - former Director of the National Institutes of Health, Francis Collins, MD, PhD, during his recognition speech at #ASHG25

🤗 Out now @naturemedicine.bsky.social results of our genomic NBS study BabyScreen+ 👶🧬 👉 www.nature.com/articles/s41... 1,000 babies WGS using existing cards 600+ conditions 13 day TAT 16 diagnoses (vs 1 in std NBS) High clinical impact High parental acceptability

Parents of children with rare diseases ask: How long until our CRISPR miracle? www.statnews.com/2025/10/09/c... via @statnews.com

These Patients Got the Cure. Then It Went Away. Gene therapies for rare diseases are frequently developed then discarded by drug companies because they can’t afford to produce the treatment for more patients. www.nytimes.com/2025/09/22/h...

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...

🚨🚨🚨 Finally out!!, Our study describing the development and deployment of a bespoke CRISPR–Cas9 base editor for palliation of a severe monogenic vasculopathy by genome editing is published today in Nature Biomedical Engineering 🧵 to follow Here is a free link: rdcu.be/eFAEz

NASA Rover Findings ‘Could be the Clearest Sign of Life’ on Mars www.nytimes.com/2025/09/10/s...

Are A.I. Tools Making Doctors Worse at Their Jobs? www.nytimes.com/2025/08/28/w...

Our latest research is out today on ‪@medrxivpreprint.bsky.social: www.medrxiv.org/content/10.1... Saturation genome editing of BRCA1 across cell types accurately resolves cancer risk. Led by the amazing Phoebe Dace. This one’s packed full of data, so check out the paper. Quick highlights… 🧵 1/n

Once a Death Sentence, This Heart Condition Is Finally Treatable www.nytimes.com/2025/08/04/w...

Congrats to @ahrensnicklas.bsky.social for being named Rad Girl of the Year at the 2025 Rad Awards! Dr. Ahrens-Nicklas was nominated for her groundbreaking work in personalized gene therapy with baby KJ. Learn more about the 2025 Rad Award winners at thephiladelphiacitizen.org/2025-rad-awa...

🧬 NOW OPEN: Registration for Breakthroughs in Targeted In Vivo Gene Editing! On Nov. 20-21, dive into cutting-edge solutions for targeted delivery of #geneediting technology in vivo in San Diego and virtually. www.asgct.org/events/break...

This is a new image from JWST. The six-pointed features are stars in the Milky Way. Everything else is a galaxy. Everything else is a galaxy.

🚨🚨🚨Big news for development of in vivo CAR T cells: AbbVie (ABBV) to Acquire Capstan Therapeutics for $2.1 Billion @abbvie @capstantx #TCell #tcellrx #immunotherapy www.streetinsider.com/Corporate+Ne...

Look at those two spiral galaxies. Two Milky Ways. Two hundreds of billions of stars and planets. Two homes. *slams fists onto table* DAMMIT the universe is SO COOL.

People With Severe Diabetes Are Cured in Small Trial of New Drug www.nytimes.com/2025/06/20/h...

A Cutting-Edge Cancer Therapy Offers Hope for Patients With Lupus www.nytimes.com/2025/06/18/w...

Should We Test Babies for Incurable Diseases? www.nytimes.com/2025/06/05/h...

International Call for a 10-Year Moratorium on Heritable Human Genome Editing - Cytotherapy www.isct-cytotherapy.org/article/S146...

David Liu @harvard.edu beautifully articulates the criticality of basic science funding for developing revolutionary therapeutics like life-saving base editors 👏 youtu.be/8YhJM6zxYDw?...

I am deeply honored and humbled to have received the 2025 Merkin Prize in Biomedical Technology along with my long term mentor and colleague Carl June, and Isabelle Rivière, and Michel Sadelain, w/ collaborative work of many scientists, clinicians, & patients. www.broadinstitute.org/news/2025-me...

Robert “Ed” Smylie, the NASA official who saved the Apollo 13 crew in 1970, has died at 95. He cobbled together an apparatus made of cardboard, plastic bags and duct tape after an explosion crippled the spacecraft as it sped toward the moon. nyti.ms/3Sbusb3

STAT's @jasonmast.bsky.social is at ASGCT, where first-in-human prime editing data debuted today. Read his dispatch here and follow along for more from New Orleans over the next two days. www.statnews.com/2025/05/14/a...

@alliancerm.bsky.social @isctglobal.bsky.social @asgct.bsky.social Therapy and experts call for decade-long ban on germline gene editing @endpts.com endpts.com/advocacy-gro...

This Thursday at the #ASGCT in New Orleans: Sadik Kassim (Danaher), Kiran Musunuru (Penn) and yours truly will chart a fundamentally new map to #CRISPR cures. See you at 830a (Room 291) and at 10a (Hall F). Image: Mary Cassatt "The Map" (1890)

I am moved by news of the upcoming film about Rosalind Franklin. I have a Jewish great-grandmother, Marie Frenkel, who died in her late 30s of ovarian cancer. I can see her haplotypes in my DNA (see below) and often think of her and Franklin (who suffered the same fate). deadline.com/2025/05/nata...

#CRISPR isn't living up to its potential. Thousands of rare diseases that could be treated with gene editing remain untouched by drug companies. Two scientists, @urnov.bsky.social and @kiranmusunuru.bsky.social, have a plan to fix that. Read more in my story @endpts.com: endpts.com/crispr-isnt-...

Y'ALL. Check out this *stunning* new 3D visualization of the breathtaking Carina Nebula 🤩 I will be forever grateful that I'm the lucky human who got to reveal this image to the world in 2022. It made me cry when I first saw it. The universe is beautiful; take it in❤️ www.youtube.com/watch?v=UFaZ...

Treating the untreatable - "Enhanced CAR T-Cell Therapy for Lymphoma after Previous Failure" @pennmedicine.bsky.social @nejm.org www.nejm.org/doi/full/10....

These boxes are not moving. A mind-bending optical illusion by Japanese artist Jagarikin.

This is a new image from #JWST. The bright points with spikes are stars in the Milky Way. Everything else is a galaxy. Everything. Else. Is. A. Galaxy.

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...

Check out this recent article on a team of SCGE researchers working on a gene therapy for prion disease. 🧬🧪 news.harvard.edu/gazette/stor...

A VLP system for the delivery of various editors is optimized for the fraction of fully assembled editor ribonucleoproteins. This increases the per-VLP efficacy of editing in primary T lymphocytes and mouse models of inherited retinal disease. www.cell.com/cell/fulltex... #NBTHighlight

Biggest commitment to a 3 second joke I've ever seen

“To me, slashing funding and people from science in the United States is like burning your seed corn. It’s not even eating your seed corn. It’s just destroying it,” www.theguardian.com/science/2025...

I am pleased to share this @ahajournals.bsky.social CircGen paper. It is a team effort. I hope we have provided valuable insights on how KCNH2 variant that alters splicing and causes a large in-frame deletion can lead to a dominant negative effect of Kv11.1 K+ #ionchannel function.

I first taught undergraduate biochemistry as a GSI at Brown in 1992 - Michaelis-Menten kinetics was on the first class. I find it important that an analytical framework as central to biochemistry as Euclidian postulates are to geometry was co-developed by a woman scientist. #WomenInStem

CRISPR companies have not fared well in recent years, despite high hopes for the technology. The problem might not be one of efficacy or science — but of how investors think about the technology, @kiranmusunuru.bsky.social tells @jasonmast.bsky.social at #STATBreakthrough.

This is what Firefly's Blue Ghost lander saw as the Sun set on Sunday. The Sun is the bright lightsource at the horizon. At top is Earth (you can't quite tell it's gibbous). And that bright star between the two is Venus. I have never seen a photo of Venus from the surface of the Moon before.

This is what a lunar eclipse looks like... from the surface of the Moon. That dark circle is Earth. Firefly Aerospace's Blue Ghost lander took this image last night.

New preprint! We worked with @msftresearch.bsky.social and @broadinstitute.org to see whether large language models (LLMs) can be useful to variant scientists in deciding whether genetic variants seen in a patient are responsible for their disease. tl;dr yes they can: www.biorxiv.org/content/10.1...

Mutated DNA Restored to Normal in Gene Therapy Advance www.nytimes.com/2025/03/10/h...

Emily Whitehead's life was saved by an "academic" team at Penn Medicine - this yielded an approved therapy, Kymriah, since used by Novartius to treat ~20,000 patients with cancer. = science at research universities saves lives. Pls read second subunit below. www.cnn.com/2025/03/07/h...