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翻訳と辞書　辞書検索 [ 開発暫定版 ] スポンサード リンク LentiGlobin BB305 ： ウィキペディア英語版 LentiGlobin BB305 LentiGlobin BB305 is an experimental treatment for beta thalassemia, a rare and potentially debilitating blood disorder. It is being developed by Bluebird Bio and was given “breakthrough therapy” designation by the Food and Drug Administration in February, 2015. In early clinical trials several patients with beta thalassemia, who usually require frequent blood transfusions to treat their disease, were able to forgo blood transfusions for extended periods of time.〔(8 December 2014) (bluebird bio Announces Data Demonstrating First Four Patients with β-Thalassemia Major Treated with LentiGlobin™ are Transfusion-Free ) Yahoo News, Retrieved 17 May 2015〕 ==Mechanism of action== Beta thalassemia is caused by mutations to or deletions of the HBB gene leading to reduced or absent synthesis of the beta chains of hemoglobin that result in variable outcomes ranging from severe anemia to clinically asymptomatic individuals. LentiGlobin BB305 is a lentiviral vector which inserts a functioning version of the HBB gene into a patient's blood producing hematopoietic stem cells (HSC) ex vivo. The resulting engineered HSC cells are then reintroduced to the patient. 抄文引用元・出典: フリー百科事典『 ウィキペディア（Wikipedia）』 ■ウィキペディアで「LentiGlobin BB305」の詳細全文を読む スポンサード リンク 翻訳と辞書 : 翻訳のためのインターネットリソース Copyright(C) kotoba.ne.jp 1997-2016. All Rights Reserved.