Gene therapy for chronic granulomatous disease.
Chronic granuloniatous disease (CGD) is a rare inherited imnunodeficiency characterized by recurrent, often life threatening bacterial and fungal infections due to a functional defect in the microbial-killing activity of phagocytic neutrophils. If regular care and conventional therapy fail, tile disease can be cured by bone marrow transplantation. This treatment is, however, only available to patients with human leukocyte antigen-identical sibling or matched unrelated donors. One therapeutic option for patients lacking suitable donors is the genetic modification of autologous hematopoietic stem cells. This review discusses the developments that have led to the realization of a successful gene therapy protocol for the correction of CGD.
Stein
Stefan
S
Institute for Biomedical Research, Georg-Speyer-Haus, Paul-Ehrlich-Strasse 42-44, 60596 Frankfurt, Germany.
Siler
Ulrich
U
Ott
Marion G
MG
Seger
Reinhard
R
Grez
Maunel
M
D016026:Bone Marrow Transplantation / Q000379:methods; D015316:Genetic Therapy / Q000379:methods*; D005822:Genetic Vectors / Q000235:genetics; D006105:Granulomatous Disease, Chronic / Q000628:therapy*; D006801:Humans; D012190:Retroviridae / Q000235:genetics