基因疗法治疗“泡泡男孩”病。
Gene Therapy for "Bubble Boy" Disease.
机构信息
Cancer Center and Center for Transplantation Sciences, Massachusetts General Hospital, Boston, MA 02129, USA.
出版信息
Cell. 2016 Jul 14;166(2):263. doi: 10.1016/j.cell.2016.06.049.
PMID:27419862
Abstract
Adenosine deaminase (ADA) deficiency results in the accumulation of toxic metabolites that destroy the immune system, causing severe combined immunodeficiency (ADA-SCID), often referred to as the "bubble boy" disease. Strimvelis is a European Medicines Agency approved gene therapy for ADA-SCID patients without a suitable bone marrow donor.
摘要
腺苷脱氨酶（ADA）缺乏会导致有毒代谢物的积累，破坏免疫系统，导致严重联合免疫缺陷（ADA-SCID），通常被称为“泡泡男孩”病。Strimvelis 是一种获得欧洲药品管理局批准的基因疗法，适用于没有合适骨髓供体的 ADA-SCID 患者。
Zotero 插件