TY - CHAP M1 - Book, Section TI - Gene Therapy for Hematologic Diseases A1 - Fung, Hua A1 - Gerson, Stanton A2 - Kaushansky, Kenneth A2 - Lichtman, Marshall A. A2 - Prchal, Josef T. A2 - Levi, Marcel M. A2 - Press, Oliver W. A2 - Burns, Linda J. A2 - Caligiuri, Michael Y1 - 2015 N1 - T2 - Williams Hematology, 9e AB - SUMMARYThe term gene therapy describes treatment resulting from expression of a transferred gene (or transgene) in diseased or other cells by engineered vectors. Once within the cell, the transgene can direct synthesis of a therapeutic protein that can complement a genetic deficiency or confer upon the cell a desired phenotype or function. Many clinical trials have involved gene therapy for patients with various gene-deficient hematologic diseases, such as severe combined immunodeficiency, hemophilia, Wiskott-Aldrich syndrome, chronic granulomatous disease, aplastic anemia, hemoglobinopathies, HIV infection, and leukemia. Results from some clinical trials indicate that gene therapy can cure or improve many inherited or acquired hematologic disorders. This chapter reviews the basic principles of gene transfer and the results of selected preclinical and clinical studies. SN - PB - McGraw-Hill Education CY - New York, NY Y2 - 2024/03/29 UR - hemonc.mhmedical.com/content.aspx?aid=1121091086 ER -