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J. Rädler

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Aug 2026

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

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.

Xiuming Liang, Houze Zhou, Z. Nizamudeen et al. · 0 citations