Abstract：
UNASSIGNED: Since its discovery in the early 1900s, sickle cell disease (SCD) has contributed significantly to the scientific understanding of hemoglobin and hemoglobinopathies. Despite this, now almost a century later, optimal medical management and even curative options remain limited. Encouragingly, in the last decade, there has been a push toward advancing the care for individuals with SCD and a diversifying interest in options to manage this disorder.
UNASSIGNED: Here, we review the current state of disease modifying therapies for SCD including fetal hemoglobin inducers, monoclonal antibodies, anti-inflammatory modulators, and enzyme activators. We also discuss current curative strategies with specific interest in transformative gene therapies.
UNASSIGNED: SCD is a chronic, progressive disease that despite a century of clinical description, only now is seeing a growth and advance in therapeutic options to improve the lifespan and quality of life for individuals with SCD. We anticipate newly designed and even repurposed therapies that may work as a single agent or combination agents to tackle the progression of SCD. The vast majority of individuals living with SCD are unlikely to receive gene therapy, therefore improved disease management is critical even for those that may ultimately chose to pursue a potentially curative strategy.
UNASSIGNED: Here, we review the current state of disease modifying therapies for SCD including fetal hemoglobin inducers, monoclonal antibodies, anti-inflammatory modulators, and enzyme activators. We also discuss current curative strategies with specific interest in transformative gene therapies.
UNASSIGNED: SCD is a chronic, progressive disease that despite a century of clinical description, only now is seeing a growth and advance in therapeutic options to improve the lifespan and quality of life for individuals with SCD. We anticipate newly designed and even repurposed therapies that may work as a single agent or combination agents to tackle the progression of SCD. The vast majority of individuals living with SCD are unlikely to receive gene therapy, therefore improved disease management is critical even for those that may ultimately chose to pursue a potentially curative strategy.
摘要：
■自1900年代初被发现以来，镰状细胞病（SCD）对血红蛋白和血红蛋白病的科学理解做出了重要贡献。尽管如此,现在将近一个世纪后，最佳的医疗管理，甚至治疗选择仍然有限。令人鼓舞的是，在过去的十年里，人们一直在推动对SCD患者的护理,并对治疗该疾病的选择产生了多样化的兴趣.
■这里，我们回顾了SCD包括胎儿血红蛋白诱导剂在内的疾病改善疗法的现状，单克隆抗体，抗炎调节剂，和酶活化剂。我们还讨论了目前对转化基因疗法有特殊兴趣的治疗策略。
■SCD是一种慢性疾病，尽管有一个世纪的临床描述，直到现在，在改善SCD患者的寿命和生活质量的治疗选择方面才出现增长和进步。我们预计新设计的，甚至是重新利用的疗法，可以作为单一药物或组合药物来解决SCD的进展。绝大多数患有SCD的人不太可能接受基因疗法，因此,即使对于那些最终可能选择寻求潜在治愈策略的患者,改善疾病管理也是至关重要的.
■这里，我们回顾了SCD包括胎儿血红蛋白诱导剂在内的疾病改善疗法的现状，单克隆抗体，抗炎调节剂，和酶活化剂。我们还讨论了目前对转化基因疗法有特殊兴趣的治疗策略。
■SCD是一种慢性疾病，尽管有一个世纪的临床描述，直到现在，在改善SCD患者的寿命和生活质量的治疗选择方面才出现增长和进步。我们预计新设计的，甚至是重新利用的疗法，可以作为单一药物或组合药物来解决SCD的进展。绝大多数患有SCD的人不太可能接受基因疗法，因此,即使对于那些最终可能选择寻求潜在治愈策略的患者,改善疾病管理也是至关重要的.
