Today in @ScienceTM, we report an in vivo prime editing strategy that corrects a disease-causing mutation and rescues phenotypes in a mouse model of citrullinemia type 1 (CTLN1), a devastating and often lethal liver disease. https://t.co/zuviDjvaSH (1)
Excited to share our new work with the Häberle lab where we used RNA–LNP-mediated in vivo prime editing to correct a urea cycle mouse model. 3 doses of RNA-LNP normalized ammonia and citrulline levels, rescued survival, and restored ureagenesis. https://t.co/lYsjxALHIU
Excited to share our new work with the Zeilhofer lab published in @ScienceTM where we used AAV-mediated prime editing to correct a pathogenic SCN1A mutation causing GEFS+ in mice. Treatment rescued survival and reduced febrile seizures to WT levels. https://t.co/5oBuXcEEj5
Excited to share our new paper!
We used in vivo prime editing to install the Adrb1 A187V “short-sleep” variant in the mouse brain. This reshaped sleep architecture, increased exploration and memory, and restored REM sleep in an Alzheimer’s disease model. https://t.co/R2BRgQEpfR
We’re hiring! Join our in vivo immuno-engineering team @UZH_en
🔬 RNA–LNP Research Associate
🧬 Senior Research Associate (In vivo Oncology)
Apply👇
https://t.co/fFGjndzPQE
https://t.co/wPk2Q9QLqa
Led by @sharan_janjuha with @AcuitasTx@pardi_lab, our Nat Biomed Eng paper is out 🧬
We use in situ sequencing to read base & prime editing directly in tissues - from mouse brain (AAV) to macaque liver (RNA–LNP)-mapping lobules & redosing. Open access: https://t.co/uhohboUWnb
Excited to share our new protocol in #NatureProtocols! 🧬👨💻🎉
A comprehensive guide for using PRIDICT2.0 and ePRIDICT machine learning tools to design efficient #CRISPR-Cas9 prime editing experiments. 🧵
@UZH_Science@schwanklab@krauthammerlab
https://t.co/xsIu9WFfHi
Congrats to the entire team and collaborators @uzh @AcuitasTx@KispiZuerich@ETH@upenn. Step-by-step we are getting closer to clinical translation of prime editing to treat metabolic liver diseases!
Have you ever wanted to design protein binders with ease? Today we present 𝑩𝒊𝒏𝒅𝑪𝒓𝒂𝒇𝒕, a user-friendly and open-source pipeline that allows to anyone to create protein binders de novo with high experimental success rates. @befcorreia@sokrypton
https://t.co/IPhMFpRgHh
Excited to share our latest study!
Today in @naturemethods the @schwanklab and @krauthammerlab report in @marquark et al. 'Effective genome editing with an enhanced ISDra2 TnpB system and deep learning-predicted ωRNAs' (1/6)
https://t.co/lqDc5Kn2Xw
6) To apply TnpBmax in vivo we delivered it together with a PCSK9-targeting ωRNA via AAV9 into mice. This led to a robust PCSK9 knockout in the liver and a reduction in LDL-cholesterol levels