In this review, University Distinguished Professor of pharmaceutical sciences and chemical engineering Mansoor Amiji, with co-author Dimitrios Bitounis, discuss “a novel class of non-viral lipid nanoparticle (LNP)-based formulations for the in vivo delivery of genetic therapies to HSC [haematopoietic stem cells] in the bone marrow with the potential to treat blood disorders,” which have the potential to overcome clinical risks that “require removal of the patients own HSC, their curative modification, and transplantation back to the patient.”