Introducing PERCEPT, a non-viral CRISPR delivery platform for in vivo genome editing! We reversibly attach peptides and polymers to CRISPR enzymes, driving editing in the brain, retina, muscle, or lungs of mice via local delivery. 🧵below, bioRxiv🔗https://t.co/KwJi88g3la
Massive credit goes to Chris Baehr @baehr_chris, who led this work, as well as Alzbeta Ressnerova, Min Kang, Sheng Zhao, Beatrice Le, Rania Haddad & Kunica Asija; Niren Murthy, John Flannery, Victor Van Laar, and Russ Lonser... and all our collaborators!
Introducing PERCEPT, a non-viral CRISPR delivery platform for in vivo genome editing! We reversibly attach peptides and polymers to CRISPR enzymes, driving editing in the brain, retina, muscle, or lungs of mice via local delivery. 🧵below, bioRxiv🔗https://t.co/KwJi88g3la
A central finding: the best delivery chemistry (which defines PERCEPT) depended on the tissue. P55 + PEG performed best in lung, while S315 + PEG performed best in muscle. PERCEPT turns protein surface chemistry into a modular toolkit for expanding the reach of genome editing.
@Ella_Maru Love this! Scariest thing in our lab is... whatever causes our E. coli cultures to collapse for a month, only to vanish as mysteriously as it appeared. Probably phage?
@chandnirao_here@AnthropicAI@DarioAmodei@mgdurrant Your perspective is spot-on IMO. I didn't think you were suggesting that the citation was missing - that's my own curiosity. It seems very odd that the Korn citation appears only at the end of the manuscript. Typically that would be prominently cited when introducing the locus.
🧬 Can CRISPR dial down α-synuclein instead of knocking it out?
MTI winners Ross Wilson & Hanqin Li aim to halt Lewy bodies with precision base editing - $90 K + full platform support.
#CRISPR#Parkinsons#MTIAwards
🔗 Read more at https://t.co/7hZ8tSvWkz
Inaugural post! (after a long hiatus)
Last May I quit my job to build @dittobio with @Mezarque and @emilycpierce ! We turn parasite molecules into drugs for autoimmune disease, and are part of the @ycombinator W26 batch.
Thanks for betting on us @agupta
Big news! Our team developed Neuro-PERC, a non-viral CRISPR delivery platform for genome editing of the brain. In our new preprint - https://t.co/8CL6LIs2Bu - we report efficient editing in multiple models & species, including field-first results in large animals (pigs) 🧵👇
@jacksonlab@Mizzou Together, these findings establish Neuro-PERC as a versatile, scalable, well-tolerated platform… as well as a promising candidate for clinical translation in neurotherapeutics. 🐭🐷🧠🧬✂️