So gene therapies are fantastic for diseases that can be attributed to a single gene mutation or single root cause.
因此,对于可归因于单一基因突变或单一根本原因的疾病,基因疗法非常有效。
TED演讲(音频版) 2021年10月合集
There is clear evidence that a single gene mutation can lead to obesity, such as, for instance, leptin deficiency and POMC deficiency.
有明确的证据表明,单个基因突变会导致肥胖,例如瘦素缺乏和 POMC 缺乏。
英语百科
Genetic disorder
A genetic disorder is a genetic problem caused by one or more abnormalities in the genome, especially a condition that is present from birth (congenital). Most genetic disorders are quite rare and affect one person in every several thousands or millions.