Non-Therapeutic Protocol

EPSTRV: Experimental Evaluation of the Potential to Correct the Pathophysiology of Sickle Cell Anemia or Thalassemia by Retroviral Vector Mediated Globin Gene Transfer

Type of Protocol/Clinical Study

Associated Hematology Studies : Sickle cell anemia


Sickle cell anemia is an inherited disease primarily affecting individuals of African American descent.  It can both shorten and reduce the quality of life by creating risk for strokes and causing chronic organ damage.

Gene therapy may potentially address the lack of available healing therapy.  This study will use bone marrow stem cells from patients with sickle cell disease or thalassemia.  Stem cells are able to become any type of blood cell.  They usually live in the bone marrow.  Participants will be asked to donate stem cells and have them collected by a procedure known as bone marrow aspiration.



Only current St. Jude patients are eligible.

Principal Investigator

Arthur Nienhuis, MD

The above information is intended to provide only a basic description about a research protocol that may be currently active at St. Jude. The details made available here may not be the most up-to-date information on protocols used by St. Jude. To receive full details about a protocol and its status and or use at St. Jude, a physician must contact St. Jude directly.