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| Item Type: | Article |
|---|---|
| Title: | Advanced peptide nanoparticles enable robust and efficient delivery of gene editors across cell types |
| Creators Name: | Gustafsson, Oskar, Krishna, Supriya, Borate, Sophia, Ghaeidamini, Marziyeh, Liang, Xiuming, Saher, Osama, Cuellar, Raul, Birdsong, Björn K, Roudi, Samantha, Estupiñán, H Yesid, Alici, Evren, Smith, C I Edvard, Esbjörner, Elin K, Spuler, Simone, de Jong, Olivier Gerrit, Escobar, Helena, Nordin, Joel Z and Andaloussi, Samir E L |
| Abstract: | Efficient delivery of the CRISPR/Cas9 system and its larger derivatives, base editors, and prime editors remain a major 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 an important advancement in the feasibility and scalability of gene-editing delivery technologies. |
| Keywords: | Synthetic Gene Editor Delivery, Protein Delivery, Cell-Penetrating Peptide (CPP), Diverse Cells, Including MuSC and iPSC, Gene Editing, Base and Primer Editor |
| Source: | Journal of Controlled Release |
| ISSN: | 0168-3659 |
| Publisher: | Elsevier |
| Volume: | 386 |
| Page Range: | 114038 |
| Date: | 10 October 2025 |
| Official Publication: | https://doi.org/10.1016/j.jconrel.2025.114038 |
| PubMed: | View item in PubMed |
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