Malech Harry L, Hickstein Dennis D
Laboratory of Host Defenses, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, Maryland 20982-1456, USA.
Curr Opin Hematol. 2007 Jan;14(1):29-36. doi: 10.1097/00062752-200701000-00007.
PURPOSE OF REVIEW: Chronic granulomatous disease and leukocyte adhesion deficiency are the major primary immune deficiencies affecting phagocytic blood cells. Major advances in clinical diagnosis and development of novel treatments for these disorders merit review. RECENT FINDINGS: Clinically beneficial gene therapy correction of X-linked chronic granulomatous disease in two adult patients was reported. Nonmyeloablative busulfan conditioning before administration of gene corrected autologous hematopoietic stem cells was likely an essential maneuver to achieve successful gene therapy. There is an increased association of autoimmune disorders with chronic granulomatous disease. Preimplantation genetic diagnosis of leukocyte adhesion deficiency-I led to the birth of a normal child. A canine model of leukocyte adhesion deficiency-I facilitated development of new nonmyeloablative hematopoietic stem cell transplant and gene therapy approaches to leukocyte adhesion deficiency. Nonmyeloablative transplantation may provide an effective, but less toxic approach for leukocyte adhesion deficiency in children. There have been advances in understanding the basis of leukocyte adhesion deficiency-II and III. SUMMARY: The most important subjects reviewed in this chapter include new advances in development of gene therapy for chronic granulomatous disease and leukocyte adhesion deficiency-I; transplantation for leukocyte adhesion deficiency-I; prenatal diagnosis of leukocyte adhesion deficiency-I; and association of autoimmune diseases with chronic granulomatous disease.
Allergy Asthma Immunol Res. 2014-1-20
Curr Opin Hematol. 2013-1
Physiology (Bethesda). 2011-10