Harvard Medical School, Boston, MA 02115, USA.
Hematol Oncol Clin North Am. 2010 Dec;24(6):1005-20. doi: 10.1016/j.hoc.2010.08.009.
The thalassemias are attributable to the defective production of the α- and β-globin polypeptides of hemoglobin. Significant discoveries have illuminated the pathophysiology and enhanced the prevention and treatment of the thalassemias, and this article reviews many of the advances that have occurred in the past 50 years. However, the application of new approaches to the treatment of these disorders has been slow, particularly in the developing world where the diseases are common, but there is definite progress. This article emphasizes how the increasing knowledge of cellular and molecular biology are facilitating the development of more effective therapies for these patients.
地中海贫血是由于血红蛋白的α-和β-球蛋白多肽生产缺陷所致。重大发现阐明了地中海贫血的病理生理学，并增强了对其的预防和治疗，本文综述了过去 50 年来发生的许多进展。然而，这些疾病在发展中国家很常见，但新的治疗方法的应用却一直进展缓慢，特别是在发展中国家。本文强调了细胞和分子生物学知识的不断增加如何促进为这些患者开发更有效的治疗方法。
