So proud to present this work today— a project spanning three PhDs and over 7 years of close collaboration. I’m thrilled to have been able to work with such a talented and devoted team.
Today we report in @NatureMedicine 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)
https://t.co/jYVDj7JPBZ
The Raguram Lab is recruiting at all levels! If you’re excited about studying intercellular biomolecule transport and developing new protein delivery technologies, apply to join our team @WhiteheadInst. Learn more here: https://t.co/s3Ko2qM0t7
Huge thanks to the entire team who made this work possible & a special thanks to my co-first @samagyabanskota ! Excited to see how the community uses these tools 🧰 🧬
Today we report in @NatureBiotech the development of dual-AAV in vivo delivery systems for prime editors that can support efficient prime editing in multiple organs such as brain, liver, and heart.
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Today we report in @natBME the development of a suite of adenine based editors (ABEs) that are small enough to each fit, together with a guide RNA & all needed regulatory elements, into a single AAV to support efficient base editing in animals. 1/4
https://t.co/PLGg1b2Oce
Today we report in @CellCellPress an engineered virus-like particle (eVLP) platform that mediates efficient in vivo delivery of therapeutic proteins, including base editors and Cas9 nuclease, resulting in therapeutic editing in multiple mouse organs. 1/14
https://t.co/v1yBl296L6
Today we report in @Nature a base editing strategy that efficiently corrects the mutation in the β-globin gene HBB that causes sickle cell disease (SCD), the most common deadly genetic disorder, rescuing the disease in mice. https://t.co/Gj8VOAFrc1
https://t.co/gzO3WwNuKT
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