Related Experiment Video
Updated: Oct 3, 2026

08:32
CRISPR/Cas9 Gene Editing of Hematopoietic Stem and Progenitor Cells for Gene Therapy Applications
Published on: August 9, 2022
Editing cells in place: Targeted in vivo gene editing of hematopoietic stem cells
Sijin Luozhong1, Angelica Varesi2, Bowen Li3
1Leslie Dan Faculty of Pharmacy, University of Toronto, Toronto, ON M5S 3M2, Canada.
Cell Stem Cell
|October 1, 2026
Abstract:
Traditional ex vivo hematopoietic stem cell (HSC) gene therapy faces manufacturing challenges. A recent study1 in Nature Biomedical Engineering demonstrates that antibody-engineered lipid nanoparticles achieve efficient and durable in vivo editing of HSCs in humanized mice, offering a promising therapeutic strategy for genetic blood diseases.

