Michael Smith Laboratories, University of British Columbia, Vancouver, British Columbia, Canada.
Centre for Blood Research, University of British Columbia, Vancouver, British Columbia, Canada.
Transfusion. 2021 Jul;61 Suppl 1:S275-S285. doi: 10.1111/trf.16519.
Platelet transfusions are an integral component of balanced hemostatic resuscitation protocols used to manage severe hemorrhage following trauma. Enhancing the hemostatic potential of platelets could lead to further increases in the efficacy of transfusions, particularly for non-compressible torso hemorrhage or severe hemorrhage with coagulopathy, by decreasing blood loss and improving overall patient outcomes. Advances in gene therapies, including RNA therapies, are leading to new strategies to enhance platelets for better control of hemorrhage. This review will highlight three approaches for creating modified platelets using gene therapies: (i) direct transfection of transfusable platelets ex vivo, (ii) in vitro production of engineered platelets from platelet-precursor cells, and (iii) modifying the bone marrow for in vivo production of modified platelets. In summary, modifying platelets to enhance their hemostatic potential is an exciting new frontier in transfusion medicine, but more preclinical development as well as studies testing the safety and efficacy of these agents are needed.
血小板输注是平衡止血复苏方案的一个组成部分，用于治疗创伤后严重出血。通过减少失血和改善整体患者预后，可以提高血小板的止血潜力，从而进一步提高输血的疗效，尤其是对于非可压缩性躯干出血或伴有凝血功能障碍的严重出血。基因治疗的进展，包括 RNA 治疗，正在为增强血小板以更好地控制出血带来新的策略。这篇综述将重点介绍三种使用基因治疗来创建改良血小板的方法：（i）体外直接转染可输注的血小板，（ii）从血小板前体细胞体外生产工程化血小板，以及（iii）修饰骨髓以在体内产生改良血小板。总之，修饰血小板以增强其止血潜力是输血医学的一个令人兴奋的新领域，但需要更多的临床前开发以及这些药物的安全性和有效性的研究。
