Alexander Astrakhan

Sasha Astrakhan

Department: Immunology
Year Entered: 2005
Prior Degrees: BS, University of North Carolina, Chapel Hill, 2002

Research Synopsis:
Wiskott-Aldrich Syndrome is a rare and typically fatal immunodeficiency characterized by a blunted immune response, eczema and increased bleeding. Patients with this disease have a very poor prognosis in the absence of a bone marrow donor; however bone marrow transplantation is not a viable option for a number of patients lacking suitable donors. Gene therapy offers an alternative approach for the treatment of patients who lack suitable bone marrow donors. My research focuses on developing a pre-clinical model for gene therapy treatment of Wiskott-Aldrich Syndrome. This approach corrects the genetic defect in stem cells isolated from the patient, and the stem cells are then re-introduced into the patient. If successful, the treated stem cells expand and differentiate into white blood cells, resulting in correction of disease symptoms. We hope to eventually translate this research to the clinic for the initiation of clinical trials for gene therapy treatment of Wiskott-Aldrich Syndrome.

Why I am participating in the Molecular Medicine Program:
I joined the molecular medicine program for the opportunity to interact with patients and observe how research findings are applied in the clinic. This experience has been invaluable for my understanding of the scientific, legal and ethical barriers for successful translation of basic research into clinical treatments.

Publications:
Astrakhan, A., Ochs, H.D., and Rawlings, D.J. 2009. Wiskott-Aldrich syndrome protein is required for homeostasis and function of invariant NKT cells. J Immunol 182: 7370-7380.

Yu PW, Tabuchi RS, Kato RM, Astrakhan A, Humblet-Baron S, Kipp K, Chae K, Ellmeier W, Witte ON, Rawlings DJ 2004. Sustained correction of B-cell development and function in a murine model of X-linked agammaglobulinemia (XLA) using retroviral-mediated gene transfer. Blood 104(5):1281-90.

Astrakhan A, Omori M, Nguyen T, Becker-Herman S, Iseki M, Aye T, Hudkins K, Dooley J, Farr A, Alpers CE, Ziegler SF, Rawlings DJ. 2007. Local increase in thymic stromal lymphopoietin induces systemic alterations in B cell development. Nature Immunol. 8(5):522-31.