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LentiGlobin BB305

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LentiGlobin BB305是一種治療β-地中海貧血英语beta thalassemia的實驗性療法,β-地中海貧血是一種罕見但可能會使人衰弱的血液疾病。這項療法由Bluebird Bio開發,並在2015年2月獲美国食品药品监督管理局指定為「突破性療法」[1]。在早期的臨床試驗中,數位需要長期輸血治療的患者在此療法後能夠更長時間不用輸血[2][3][4]

作用機轉

β-地中海貧血是HBB英语HBB基因突變或缺失造成,血红蛋白的β鏈合成減少或缺乏,最終導致由嚴重貧血到無症狀等程度不等的結果[5]。LentiGlobin BB305是一種病毒載體,能在生物體外將具功能的HBB基因插入患者的造血干细胞(HSC),之後再將經基因工程改造的造血幹細胞植回患者體內[6][7]

參見

參考資料

  1. ^ Ten things you might have missed Monday from the world of business. 波士頓環球報. 3 February 2015 [13 February 2015]. (原始内容存档于2020-08-01). 
  2. ^ Cavazzana-Calvo M, Payen E, Negre O, et al. Transfusion independence and HMGA2 activation after gene therapy of human β-thalassaemia. Nature. 2010, 467 (7313): 318–22. PMC 3355472可免费查阅. PMID 20844535. doi:10.1038/nature09328. 
  3. ^ Winslow, Ron. New Gene Therapy Shows Promise for Lethal Blood Disease需要付费订阅. 华尔街日报. 8 December 2015 [13 February 2015]. (原始内容存档于2020-03-02). 
  4. ^ (8 December 2014) bluebird bio Announces Data Demonstrating First Four Patients with β-Thalassemia Major Treated with LentiGlobin™ are Transfusion-Free页面存档备份,存于互联网档案馆) Yahoo News, Retrieved 17 May 2015
  5. ^ Cao, Antonio; Galanello, Renzo. Beta-thalassemia. Genetics in Medicine. 21 January 2010, 12 (2): 61–76 [14 February 2015]. PMID 20098328. doi:10.1097/GIM.0b013e3181cd68ed. (原始内容存档于2017-06-23). 
  6. ^ Negre O, et al. Preclinical evaluation of efficacy and safety of an improved lentiviral vector for the treatment of β-thalassemia and sickle cell disease (PDF). Current Gene Therapy. 2015, 15 (1): 64–81 [2019-01-10]. PMC 4440358可免费查阅. PMID 25429463. doi:10.2174/1566523214666141127095336. (原始内容存档 (PDF)于2018-07-19). 
  7. ^ Thompson A, et al. Initial Results from the Northstar Study (HGB-204): A Phase 1/2 Study of Gene Therapy for β-Thalassemia Major Via Transplantation of Autologous Hematopoietic Stem Cells Transduced Ex Vivo with a Lentiviral βΑ-T87Q -Globin Vector (LentiGlobin BB305 Drug Product). Blood. 2014, 124 (21): 549 [2019-01-10]. (原始内容存档于2019-10-18). 

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