Potential therapeutic implications of hematopoietic stem cell gene therapy in the correction of mutationcausing human beta-thalassemia

<p>Dina A Ghoraba</p>

Potential therapeutic implications of hematopoietic stem cell gene therapy in the correction of mutationcausing human beta-thalassemia

Dina A Ghoraba

Kasr El Aini Medical School Teaching Hospitals, Egypt

: J Appl Bioinforma Comput Biol

Abstract

Hematopoietic stem cell gene therapy (HSC-GT) or in vivo gene-based autologous stem cell transplant is one of the most exciting clinical tools to emerge from the gene therapy. By using integrating vectors, therapeutic transgenes can be efficiently transmitted to all HSC-derived mature hematopoietic stem cells via mitosis. This strategy generates an alternative or complementary clinical therapy for bone marrow transplant patients with no related human-leukocyte-antigen-matched or Geno-identical donors. Successful HSC-GT clinical trials have been conducted to treat several genetic and immune diseases of the hematopoietic system as X-linked severe combined immunodeficiency (SCID-X1) and β-thalassemia. In this research we will study the effect of transfection of an erythroid promotor vector-mediated normal human β-globin gene sequence (hemoglobin subunit beta- HBB) construct to HSCs culture media from β- thalassemia patients.