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Efficient genome editing in the non-human primate brain using programmable extracellular vesicles

Xiuming Liang Houze Zhou Z. Nizamudeen Eleni Kyriakopoulou Angharad E. Evans Samantha Roudi H. Y. Estupiñán J. Rädler V. W. Hou Callum D.W. Daniel Inés Hernández-Pérez Malika Singh Pardis Parsi Daniel A. Lonergan Lidia Menéndez Berlana Robert Walmsley Ying Chen Juliette S. M. T. Suermondt M. Mowoe Andre Görgens Christopher A. Smith George O. Redrup Lewis D. Ashmore Samuel Perez Kanchan J. Thapa Shrishti Banerjee Scott E. Bonner Mariana Conceicao Rebecca L. Gavin Justin Hean Peter D. Horrocks Maria O. Levitin P. Lundin Heena Sharma R. Tawar Liang Liu Dhanu Gupta D. R. Carter J. Nordin S. E. Andaloussi
Aug 2026 · bioRxiv · 0 citations
Biology

TL;DR

It is shown that an optimized single-guide RNA scaffold architecture improves RNP stability, and when combined with additional EV engineering leads to a three-hundred-fold increase in potency, enabling efficient base editing or knockout in primary cells, human brain organoids and in vivo, including the mouse brain.

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