Namita Nabar

PhD Student
Email: nnabar [at] mit . edu

Layered lipid nanoparticles for SCD gene delivery

Recent advances in sickle cell disease therapies have identified candidate gene therapeutics for hematopoetic stem cells (HSCs). Lipid nanoparticles (LNPs) are promising nonviral vehicles for in vivo transfection, a potentially accessible and easily deployable treatment option. However, therapeutic efficacy is limited by LNPs’ ability to access and transfect HSCs in vivo. My research seeks to engineer LNP surface chemistry to increase HSC uptake, transfection, and gene expression, by screening libraries of polymer backbones and exploring cell-penetrating peptide additions.