LentiGlobin BB305

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

基因治療

參考資料

^ 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）.
^ 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）.

外部連結

Bluebird Bio介紹LentiGlobin BB305產品的網頁

[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 . 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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