Thalassemia is a group of inherited blood disorders.

地中海贫血是一组遗传性血液疾病。

Usually, patients with mild thalassemia don't need treatment, while patients with severe thalassemia are treated with blood transfusions.

通常情况下，轻地中海贫血无需治疗，而重地贫患者需输血治疗。

These are diseases like sickle cell anemia or beta thalassemia.

比如镰状胞性贫血或β地中海贫血。

With moderate alpha thalassemia, there may be golf-ball like RBCs, due to precipitated HbH molecules.

中α地中海贫血患者，由于HbH分子沉积，可见高尔夫球胞。

And alpha thalassemia is caused by mutations in the alpha genes, most commonly a gene deletion.

而α地中海贫血是由α基因变异引起的，最常见的原因是基因缺失。

If a person has two defective alpha genes, the person has alpha thalassemia minor, which causes mild symptoms.

如果有2个α基因异常，则会出现轻α地中海贫血，会引起轻微的症状。

The initial symptoms of minor and moderate alpha thalassemia are due to anemia, which include pallor, shortness of breath, and easy fatigability.

轻和中α地中海贫血的首发症状往往是贫血，表现为苍白、气促、乏力。

All right, as a quick recap: alpha thalassemia is an autosomal recessive disorder caused by deletion of alpha globin genes on chromosome 16.

得！快速回顾一下：α地中海贫血是一种常染色体隐性遗传病，由16号染色体上的α珠蛋白基因缺陷引起。

To prevent complications from an existing blood or bleeding disorder, such as sickle cell disease, thalassemia, or anaemia caused by kidney disease, haemophilia, or von Willebrand disease.

预防现有血液或出血性疾病的并发症，如镰状胞病、地中海贫血或由肾脏疾病、血友病或血管性血友病引起的贫血。

The following year, Vertex and Crispr Therapeutics, pharmaceutical companies based in Boston, Massachusetts, and Zug, Switzerland, said they would co-develop a treatment named CTX001, a treatment for two disorders: sickle-cell disease and beta thalassemia.

次年，总部位于马萨诸塞州波士顿的制药公司Vertex和总部位于瑞士楚格的Crispr Therapeutics表示，他们将共同开发一种名为CTX001的治疗方法，用于治疗镰状胞病和β地中海贫血。