All‑RNA LNPs Achieve Efficient In‑Vivo Prime Editing
Today in @NatureNano, we report an all-RNA lipid nanoparticle (LNP) system for efficient in vivo prime editing (PE). We identify bottlenecks in transient prime editor delivery, develop a workflow for LNP optimization, and use it to rescue a mouse model of phenylketonuria. https://t.co/WAH8LHAb9z 1/12
AI‑Designed PE8 Editors Boost Prime Editing Efficiency
Today in @NatureBiotech we report a new suit of PE8 prime editor proteins. PE8 variants were developed from laboratory-evolved PE6 proteins using AI-guided protein redesign. This approach combines recent advances in computational protein design and directed evolution to increase prime...
Adenine Base Editing Reverses Dravet Syndrome in Mice
Today in @ScienceTM, we report the use of in vivo adenine base editing to correct a variant causing Dravet syndrome, a severe childhood epilepsy and neurodevelopmental disorder, substantially ameliorating disease symptoms and extending lifespan in an animal model. 1/13 https://t.co/uQEwWvj94y
Breakthrough Prize Showcases Baby Gene‑Editing Miracle
The 2026 Breakthrough Prize ceremony (“the Oscars for science”) celebrated the achievements of many remarkable scientists and their students and collaborators last weekend. Here’s the video of the show: https://t.co/eCHUnDqrYX. And here’s the segment in which Anne Hathaway and Alex...
In‑vivo Base Editing Rescues Zellweger Disorder in Mice
Today in @natBME we report an in vivo base editing strategy that corrects a common disease-causing mutation and rescues pathology in a mouse model of Zellweger spectrum disorder (ZSD) and restore peroxisomal function in patient derived cells. This work highlights...
Prime-Edited Therapy Restores Immune Function in Teen
Below is the story of the first patient treated with a prime-edited therapeutic, developed by @PrimeMedicine in a trial led by Dr. Élie Haddad and his team at CHU Sainte-Justine. This teenager suffered from chronic granulomatous disease (CGD), an immunodeficiency,...