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Today we report in Nature Biomedical Engineering the eePASSIGE system, which uses evolved and engineered recombinases and prime editing to integrate large gene-sized DNA cargoes into the mammalian genome in an efficient, precise, and targeted manner. (1/13) drive.google.com/file/d/1WDMVqL…

Glad to see our works online! Kudos to all the authors, particularly @Smriti__Pandey for working together during the entire journey. It will be excited to see labs worldwide use eePASSIGE to develop new therapeutics and address basic biological questions! nature.com/articles/s4155…

On #WorldSickleCellDay, we look to researchers, like Greg Newby (Johns Hopkins Medicine), Kelcee Everette (Broad Institute), & Benjamin L. Oakes (Scribe Therapeutics) who are developing the next generation of #SCD treatments with fewer side effects: asbmb.org/asbmb-today/sc….

Congrats Alexander Sousa and Colin Hemez !!

Great place to explore science and make innovative discoveries! Proud to see my bench mate and friend Samagya now embark her new journey as a PI at BU!

So well deserved! Congrats Sunny!

A recount and update on our efforts to bring base editing to progeria patients as part of a team including Francis Collins, Sammy Basso, Mike Erdos, Leslie Gordon, @Progeria Luke Koblan, Meirui An, Xin "Daniel" Gao, and others, nicely captured by Gina Kolata in the The New York Times:

Congratulations, Victor Ambros UMass Chan Medical School and Gary Ruvkun Harvard Medical School!!! What a day for RNA!

Congrats to Victor!!! 🎉He’s one of the best scientists I've ever met—always humble, incredibly kind, and consistently taking notes in seminars. His eagerness to learn is truly inspiring!

My congratulations to UW–Madison Biochem faculty Monica Neugebauer who was named a 2024 Packard Foundation Fellow for Science and Engineering (1 of only 20 scientists nationwide!) to recognize her important work with novel enzymes & commitment to sustainable chemistry. news.wisc.edu/monica-neugeba…

Today we report in Nature Biotechnology a system for the laboratory evolution of engineered virus-like particles (eVLPs) that enables the discovery of eVLP variants with desired properties, including improved production and delivery potency. 1/13 PDF: drive.google.com/file/d/1E6Nr86…

Today we report in Nature Medicine the development of a base editing strategy for prion disease, currently a fatal and rapidly progressing neurogenerative condition with no effective treatment. @PrionAlliance DevermanLab (1/13) drive.google.com/file/d/1DVpKrz…

A good summary from Jason Mast of gene editing’s awkward adolescent phase: pimples, braces, and all. We look enviously at our older sibling, RNAi, already moved out of the house with a steady job…

Congrats again! Well deserved. Go Allen!

In a medical milestone, a customized base editor was developed, characterized in human and mouse cells, tested in mice, studied for safety in non-human primates, cleared by U.S. FDA for clinical trial use, manufactured as a complex with an LNP, and dosed into a baby with a severe,

Today in Science Magazine 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, a collaboration with Sternberg Lab. 1/13 drive.google.com/file/d/1I-UbCR…

We’re thrilled that base editing, developed by researchers in the lab of Broad member David R. Liu, is having such a profound impact in the clinic. Congratulations to former Broad postdoc Kiran Musunuru and Children's Hospital for their groundbreaking work. nejm.org/doi/full/10.10…

Another medical milestone! The first patient treated with a prime edited therapeutic shows positive clinical outcomes consistent with effective rescue of his chronic granulomatous disease (CGD), a debilitating and life-shortening primary immunodeficiency. At least 19 base editing

The CRISPR family’s most versatile member has made its medical debut: a cutting-edge gene-editing technique known as prime editing has been used to treat a person for the first time go.nature.com/4kqr3RK

In Nature Genetics we report that base editing of trinucleotide repeats (TNRs) reduces somatic repeat expansions in Huntington’s disease (HD) and Friedreich’s ataxia (FRDA)—in patient-derived cells and in vivo—a collaboration with the Mouro Pinto lab. drive.google.com/file/d/1on7kdy… 1/14