Efficient Scaling up EV‐AAVs Production via Cellular Nanoporation for Familial Hypercholesterolaemia Therapy - Yan - 2025 - Journal of Extracellular Vesicles - Wiley Online Library isevjournals.onlinelibrary.wiley.com/doi/10.1002/...

New @nejm.org One-shot gene therapy for a type of congenital deafness enabled some children to hear for the first time, and 3 of 12 kids achieved normal hearing www.nejm.org/doi/full/10.1056/NEJMoa2400521

Glad this is out- nice work by team Portugal! :)

Thanks @bkleinstiver.bsky.social . Always enjoy the ride with you scientifically. And giving your cool editing systems a “smooth ride”! ;)

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

David Corey is one of the most brilliant scientists I’ve had the pleasure of working with. Cutting funding to scientists like him is going to hurt our country in so many ways including its standing in the world as well as economically. Not to mention the health of our citizens. All for what?

A troubling moment for the next generation of scientists. UMass Chan Med School is rescinding dozens of PhD offers in biomedical sciences due to fears over federal research funding cuts. www.nbcboston.com/news/local/u...

Broad Institute represented at #Standupforscience Boston Emily Bramel from our research team with the proteosome poster 10/10

Scientific innovation is a defining characteristic of our country. Don’t let that be a thing of the past! #StandUpforScience2025

The Trump administration continues its deluge of executive orders that directly affect science and research. Everything from cancer research to the distribution of medical information is affected. 🧪 www.iflscience.com/trump-impose...

Our new publication characterizing the function of extracellular vesicle-associated AAV (EV-AAV) vectors is out! We have developed the EV-AAV platform for several years, yet there is still much to learn about the unique AAV-based system.

Have you heard the news? Addgene’s now offering AAV Packaged on Request! Read all about it in our latest blog post. blog.addgene.org/aav...

On January 10th our viral vector service is expanding to include a packaged on request option! You will be able to choose a plasmid, select a preferred serotype, and submit a request — all in one place. Want to learn more? Sign up below for email updates. info.addgene.org/pac...

We're expanding our viral vectors service to include AAV Packaged on Request — and the ordering process will look a little different! Here's what you need to know: blog.addgene.org/add...

Please see our new publication, Metabolic engineering improves transduction efficiency and downstream vector isolation by altering the lipid composition of extracellular vesicle-enclosed AAV by Espinoza et al.

Check out our Clinical Perspective on the recent clinical trials of gene therapy for treating deafness, and on its remaining limitations with delivery issues and low spectrum of cell types targeted. Together with Landegger/Reisinger/Hage/Grimm/Cederroth labs. www.cell.com/molecular-th...