Nature biotechnology
In vivo gene editing of human hematopoietic stem and progenitor cells using envelope-engineered virus-like particles.
Vladimir V Botchkarev, Sean Harrington, Matteo Stoppato, Alexander Justen, Casey Kimber, Anjali Kapuria, Keylie M Gibson, Chi-Shuen Chu, Yuanxin Xu, Kelsey Haugh, Ramya Ankala, Nathan Kipniss, Andre DeGroot, Emerson Moore, Rowena de Jesus
Published: 202510.1038/s41587-025-02915-2
Abstract
Engineered virus-like particles (VLPs) are a promising technology for in vivo gene editing of human hematopoietic stem and progenitor cells (HSPCs). Here we design and test two different VLP envelopes for human HSPC editing in vitro and in vivo. The…
Preview only. Read the full abstract at the source