The treatment consists of a modified form of the glucocerebrosidase enzyme given intravenously 该疗法主要通过对静脉血提供的葡萄糖脑苷脂酶进行修饰。
In people with gaucher disease , the body is not able to properly produce this enzyme , and the fat can not be broken down 在戈谢病患者体内,不能完全合成该酶,从而导致葡萄糖脑苷脂不能被降解。
Normally , this gene is responsible for an enzyme called glucocerebrosidase that the body needs to break down a particular kind of fat called glucocerebroside 在通常情况下,这个基因是负责制造一种叫“葡萄糖脑苷脂酶”的酶,身体需要这种酶来降解一种叫葡萄糖脑苷脂的物质。