Email: nnabar [at] mit . edu
Polymer nanoplexes for SCD gene delivery
Recent advances in sickle cell disease therapies have identified candidate gene therapeutics for hematopoetic stem cells (HSCs). Layer-by-layer polyplexes are promising nonviral vehicles for in vivo transfection, a potentially accessible and easily deployable treatment option. However, therapeutic efficacy is limited by the polyplex’s ability to transfect HSCs. My research seeks to engineer polyplex chemistry to increase HSC uptake, transfection, and gene expression, by screening libraries of polymer backbones and exploring cell-penetrating peptide additions.