Development of Hematopoietic Stem Cell-targeted LNPs through Lipid Composition Optimization - PubMed
4 hours ago
- #Hematopoietic Stem Cells
- #Lipid Nanoparticles
- #Gene Editing
- Developed hematopoietic stem cell (HSC)-targeted lipid nanoparticles (LNPs) using Bayesian optimization with functional amino lipids to improve transfection efficiency while maintaining cell viability.
- Achieved ex vivo TP53 gene editing in cord blood CD34+ cells with up to 40% on-target editing efficiency using the optimized LNPs.
- Demonstrated efficient RNA delivery into primary human monocytic leukemia cells with one LNP formulation, highlighting potential for treating hematopoietic disorders.
- Applied machine learning-guided design to advance HSC-targeted therapies, showing promise for LNP-based gene editing platforms in hereditary and malignant conditions.