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LentiGlobin BB305

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LentiGlobin BB305是一种治疗β-地中海贫血英语beta thalassemia的实验性疗法,β-地中海贫血是一种罕见但可能会使人衰弱的血液疾病。这项疗法由Bluebird Bio开发,并在2015年2月获美国食品药品监督管理局指定为“突破性疗法”[1]。在早期的临床试验中,数位需要长期输血治疗的患者在此疗法后能够更长时间不用输血[2][3][4]

作用机转

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β-地中海贫血是HBB英语HBB基因突变或缺失造成,血红蛋白的β链合成减少或缺乏,最终导致由严重贫血到无症状等程度不等的结果[5]。LentiGlobin BB305是一种病毒载体,能在生物体外将具功能的HBB基因插入患者的造血干细胞(HSC),之后再将经基因工程改造的造血干细胞植回患者体内[6][7]

参见

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参考资料

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  1. ^ Ten things you might have missed Monday from the world of business. 波士顿环球报. 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). 

外部链接

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