Towards manufactured red blood cells for the treatment of inherited anemia.
Towards manufactured red blood cells for the treatment of inherited anemia.
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DOI：
10.3324/haematol.2020.268847
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发表时间：
2021-09-01
期刊：
影响因子：
10.1
通讯作者：
Toye AM
中科院分区：
文献类型：
--
作者：
Pellegrin S;Severn CE;Toye AM
患有遗传性贫血和血红蛋白病（如镰状细胞病和β-地中海贫血）的患者通过输注红细胞（RBC）来缓解症状。其中一些患者可能具有罕见的血型类型或继续发展同种免疫反应，这可能使其难以在供体人群中获得相容的血液。体外培养的红细胞代表了一种特别有吸引力的替代方案，可以满足未满足的临床需求。然而，挑战在于从有限数量的干细胞中产生标准成人治疗剂量所需的2x 1012 RBC。在良好生产规范下，从成体干细胞生产红细胞方面取得了令人鼓舞的进展。2011年，Douay小组在一名志愿者中成功地进行了自体制造RBC的原理验证。在英国，计划进行一项试验，通过在多名志愿者中测试同种异体小剂量的实验室生长的人造红细胞，评估人造红细胞是否等同于供体中自然产生的红细胞。这篇综述讨论了红细胞培养领域的最新进展以及进一步扩大输血实践中制造红细胞生产的机会。
Patients with inherited anemia and hemoglobinopathies (such as sickle cell disease and β-thalassemia) are treated with red blood cell (RBC) transfusions to alleviate their symptoms. Some of these patients may have rare blood group types or go on to develop alloimmune reactions, which can make it difficult to source compatible blood in the donor population. Laboratory-grown RBC represent a particularly attractive alternative which could satisfy an unmet clinical need. The challenge, however, is to produce - from a limited number of stem cells - the 2x1012 RBC required for a standard adult therapeutic dose. Encouraging progress has been made in RBC production from adult stem cells under good manufacturing practice. In 2011, the Douay group conducted a successful proof-of-principle mini-transfusion of autologous manufactured RBC in a single volunteer. In the UK, a trial is planned to assess whether manufactured RBC are equivalent to RBC produced naturally in donors, by testing an allogeneic mini-dose of laboratory-grown manufactured RBC in multiple volunteers. This review discusses recent progress in the erythroid culture field as well as opportunities for further scaling up of manufactured RBC production for transfusion practice.
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影响因子：
10.1
作者：
Heideveld E;Hampton-O'Neil LA;Cross SJ;van Alphen FPJ;van den Biggelaar M;Toye AM;van den Akker E
通讯作者：
van den Akker E
影响因子：
4
作者：
Bernecker, Claudia;Ackermann, Mania;Dorn, Isabel
通讯作者：
Dorn, Isabel
影响因子：
2.9
作者：
COLLINS, AF;GONCALVESDIAS, C;OLIVIERI, NF
通讯作者：
OLIVIERI, NF
影响因子：
20.3
作者：
Huang, Nai-Jia;Lin, Ying-Cing;Lodish, Harvey
通讯作者：
Lodish, Harvey
影响因子：
7.5
作者：
Heshusius, Steven;Heideveld, Esther;van den Akker, Emile
通讯作者：
van den Akker, Emile