New Approaches for Blood Disease Treatments

Mansoor Amiji

BouvĂ©/ChE University Distinguished Professor Mansoor Amiji’s research on “Lipid Nanoparticles Target Haematopoietic Stem Cells” was published in Nature Nanotechnology.


Abstract Source: Nature

Haematopoietic stem cells (HSC) in the bone marrow have the unique ability to develop into different types of blood cells. Genetic abnormalities in HSC are the root cause of blood disorders associated with high morbidity and high mortality rates, including haematological malignancies (for example, leukaemias) and sickle cell disease. Advancements in multi-drug chemotherapy and radiotherapy have improved overall response rates of leukaemia patients, but relapses and drug resistance are an ongoing challenge. For sickle cell disease, two one-time viral gene therapies were recently approved by the United States Food and Drug Administration (FDA). These approaches, however, carry clinical risks as they require removal of the patients own HSC, their curative modification, and transplantation back to the patient. Now, writing in Nature Nanotechnology, Lian et al. propose a novel class of non-viral lipid nanoparticle (LNP)-based formulations for the in vivo delivery of genetic therapies to HSC in the bone marrow with the potential to treat blood disorders.

Related Departments:Chemical Engineering