Poster #049, UC Davis Health System
Evaluation of A Lentiviral Gene Therapy for Treating Sickle Cell Disease
Mentor: Ping Zhou, PHD
Introduction: Sickle cell disease (SCD) is an inherited blood disorder caused by a point mutation in the β-hemoglobin gene(HBB), which results in the production of abnormal hemoglobin, sickling of red blood cells, and severe systemic injury. Lentiviral gene therapy is an effective approach to cure SCD by inserting a functional copy of HBB into hematopoietic stem cells.
We hypothesized that human CD34+ hematopoietic stem cells transduced with lentiviruses carrying HBB would result in increased β -globin gene expression in their progeny of red blood cell lineage.
Method: In our study, human CD34+ hematopoietic stem cells transduced with or without a lentiviral vector carrying the therapeutic HBB were transplanted in mice. After four
months, bone marrow was collected, human CD235+ cells were isolated, RNA was extracted and converted to cDNA, and quantitative polymerase chain reaction (qPCR) was
performed to detect the β-globin gene expression.
Results: RT-qPCR analysis successfully amplified the β -globin cDNA from the isolated human CD235+ erythroid lineage cells, ensuring successful detection of Beta-globin gene expression following stem cell transplantation.