Dr. Sadelain is a geneticist working in the area of gene transfer-the notion of treating a disease by inserting a healthy gene in place of one that is malfunctioning or missing. Dr. Sadelain is trying to insert genes into bone marrow cells and T lymphocytes to reduce the risk of graft-versus-host disease, while preserving the antileukemic effect of the bone marrow transplantation. Another of Dr. Sadelain's goals is to enhance the use of T lymphocytes as therapeutic tools by targeting them to cancer cells, making sure they go where they should in the body to kill tumor cells. Sadelain is seeking to improve gene transfer and gene expression in blood-forming cells and cells of the immune system. His ultimate goal is to use gene transfer mediated by retroviruses to create improved treatments for genetic disorders, such as sickle cell anemia, and for cancer.
Chang AH, Stephan MT, Lisowski L, Sadelain M. Erythroid-specific Human Factor IX Delivery From In Vivo Selected Hematopoietic Stem Cells Following Nonmyeloablative Conditioning in Hemophilia B Mice.
Mol Ther. 2008 Aug 5. [Epub ahead of print]
Dobrenkov K, Olszewska M, Likar Y, Shenker L, Gunset G, Cai S, Pillarsetty N, Hricak H, Sadelain M, Ponomarev V. Monitoring the efficacy of adoptively transferred prostate cancer-targeted human T lymphocytes with PET and bioluminescence imaging. J Nucl Med. 2008 Jul;49(7):1162-70. Epub 2008 Jun 13.
Lisowski L, Sadelain M. Current status of globin gene therapy for the treatment of beta-thalassaemia. Br J Haematol. 2008 May;141(3):335-45. Review.