Retroviral Vector Mediated Globin Gene Transfer to Correct Sickle Cell Anemia or Thalassemia
Using sickle cell and thalassemia mouse models, researchers will evaluate the possibility of correcting these disorders by inserting healthy genetic material into the diseased blood cells. Human participants affected with sickle cell disease or thalassemia will donate bone marrow for use in the mouse models.
|Study Design:||Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
|Official Title:||Experimental Evaluation of the Potential to Correct the Pathophysiology of Sickle Cell Anemia or Thalassemia by Retroviral Vector Mediated Globin Gene Transfer|
- To develop retroviral vector mediated gene transfer as a potentially curative therapy for sickle cell anemia and β-thalassemia. [ Time Frame: 4 years ] [ Designated as safety issue: Yes ]The specific hypothesis to be tested is that a vector can be designed that achieves a therapeutic level of globin production in transduced cells.
|Study Start Date:||July 2007|
|Estimated Study Completion Date:||July 2016|
|Estimated Primary Completion Date:||July 2015 (Final data collection date for primary outcome measure)|
Genetic: Gene Therapy
Human participants affected with sickle cell disease or thalassemia will donate bone marrow for use in the mouse models
These studies are designed to evaluate the potential of retroviral vector mediated gene transfer to correct the pathophysiology of sickle cell anemia and β-thalassemia. CD34+ cells purified from bone marrow of research participants with a sickle cell syndrome or a thalassemia syndrome will be transduced with retroviral vectors containing γ-globin coding sequences under the control of the β-globin gene promoter and including various regulatory elements chosen to enhance gene expression and to insulate regulatory elements from cellular genes at or near the integration sites. The efficiency of gene transfer and the function of the globin transgene will be evaluated in erythroid cells derived from transduced progenitors and from the progenitors in the bone marrow of immunodeficient mice engrafted with transduced, primitive hematopoietic cells. This study will evaluate whether a vector can be designed to achieve both a potentially therapeutic efficiency of gene transfer into repopulating cells and a potentially therapeutic level of globin gene expression in maturing erythroid cells.
|Contact: Arthur W Nienhuis, MDemail@example.com|
|United States, Tennessee|
|St. Jude Children's Research Hospital||Recruiting|
|Memphis, Tennessee, United States, 38105|
|Contact: Arthur W Nienhuis, MD 866-278-5833 firstname.lastname@example.org|
|Principal Investigator: Arthur W Nienhuis, MD|
|Principal Investigator:||Arthur W Nienhuis, MD||St. Jude Children's Research Hospital|