Division of Allergy and Immunology, Children's National Medical Center, Washington, DC.
Laboratory of Clinical Immunology and Microbiology, National Institute of Allergy and Infectious Disease, Bethesda, Maryland.
J Pediatric Infect Dis Soc. 2018 May 9;7(suppl_1):S40-S44. doi: 10.1093/jpids/piy011.
Chronic granulomatous disease is a rare and potentially fatal disorder of neutrophil function. Beyond current medical management and hematopoietic stem cell transplantation, new methods of gene therapy that use lentiviral vectors or gene editing might extend curative therapies to patients who lack a suitable transplantation donor while eliminating the risk of graft-versus-host disease. Furthermore, new therapies focused on altering the biology of phagolysosomes might offer novel targeted treatments for inflammatory complications in patients with chronic granulomatous disease.
慢性肉芽肿病是一种罕见的、潜在致命的中性粒细胞功能障碍疾病。除了目前的医疗管理和造血干细胞移植外，使用慢病毒载体或基因编辑的新基因治疗方法可能会为缺乏合适移植供体的患者提供治愈疗法，同时消除移植物抗宿主病的风险。此外，专注于改变吞噬溶酶体生物学的新疗法可能为慢性肉芽肿病患者的炎症并发症提供新的靶向治疗。
