Thalassemia is a group of inherited blood disorders.

中海贫是一组遗性液疾病。

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

通常情况下，轻中海贫无治疗，重贫患者治疗。

These are diseases like sickle cell anemia or beta thalassemia.

比如镰状细胞性贫或β中海贫。

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

中α中海贫患者，由于HbH分子沉积，可见高尔夫球样红细胞。

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

α中海贫是由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中海贫，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中海贫的首发症状往往是贫，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中海贫是一种常染色体隐性遗病，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.

预防现有液或出性疾病的并发症，如镰状细胞病、中海贫或由肾脏疾病、友病或管性友病引起的贫。

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，用于治疗镰状细胞病和β中海贫。