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| Item Type: | Preprint |
|---|---|
| Title: | Advanced peptide nanoparticles enable robust and efficient delivery of gene editors across cell types |
| Creators Name: | Gustafsson, O., Krishna, S., Borate, S., Ghaeidamini, M., Liang, X., Saher, O., Cuellar, R., Birdsong, B.K., Roudi, S., Estupiñán, Y.H., Alici, E., Smith, E.C.I., Esbjörner, E.K., Spuler, S.K., de Jong, O.G., Escobar, H., Nordin, J.Z. and Andaloussi, S.E.L. |
| Abstract: | Efficient delivery of the CRISPR/Cas9 system and its larger derivatives, base editors, and prime editors remain a significant challenge, particularly in tissue-specific stem cells and induced pluripotent stem cells (iPSCs). This study optimized a novel family of cell-penetrating peptides, hPep, to deliver gene-editing ribonucleoproteins. The hPep-based nanoparticles enable highly efficient and biocompatible delivery of Cre recombinase, Cas9, base-, and prime editors. Using base editors, robust and nearly complete genome editing was achieved in the human cells: HEK293T (96%), iPSCs (74%), and muscle stem cells (80%). This strategy opens promising avenues for ex vivo and, potentially, in vivo applications. Incorporating silica particles enhanced the system's versatility, facilitating cargo-agnostic delivery. Notably, the nanoparticles can be synthesized quickly on a benchtop and stored as lyophilized powder without compromising functionality. This represents a significant advancement in the feasibility and scalability of gene-editing delivery technologies. |
| Source: | bioRxiv |
| Publisher: | Cold Spring Harbor Laboratory Press |
| Article Number: | 2024.11.27.624305 |
| Date: | 4 December 2024 |
| Official Publication: | https://doi.org/10.1101/2024.11.27.624305 |
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