LentiGlobin BB305

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

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

基因治疗

参考资料

^ 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）.
^ 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 . PMID 20844535. doi:10.1038/nature09328.
^ Winslow, Ron. New Gene Therapy Shows Promise for Lethal Blood Disease. 华尔街日报. 8 December 2015 [13 February 2015]. （原始内容存档于2020-03-02）.
^ (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
^ 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）.
^ 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 . PMID 25429463. doi:10.2174/1566523214666141127095336. （原始内容存档 (PDF)于2018-07-19）.
^ 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

Bluebird Bio介绍LentiGlobin BB305产品的网页

[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 . PMID 20844535. doi:10.1038/nature09328.

[3] Winslow, Ron. New Gene Therapy Shows Promise for Lethal Blood Disease. 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 . 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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