New insights into the pathogenesis of adenosine deaminase-severe combined immunodeficiency and progress in gene therapy
AV Sauer, A Aiuti - Current opinion in allergy and clinical …, 2009 - journals.lww.com
AV Sauer, A Aiuti
Current opinion in allergy and clinical immunology, 2009•journals.lww.comGene therapy for ADA-deficient SCID is an effective treatment, providing long-term clinical
benefit for affected patients. Future research will be needed to address the occurrence of
autoimmune manifestations and nonimmunological defects in order to improve patients' long-
term prospects.
benefit for affected patients. Future research will be needed to address the occurrence of
autoimmune manifestations and nonimmunological defects in order to improve patients' long-
term prospects.
Summary
Gene therapy for ADA-deficient SCID is an effective treatment, providing long-term clinical benefit for affected patients. Future research will be needed to address the occurrence of autoimmune manifestations and nonimmunological defects in order to improve patients' long-term prospects.
Lippincott Williams & Wilkins