Prasad 警告称,与简单的酶缺陷修复不同,基因疗法可能难以逆转缺陷蛋白多年累积造成的结构性损伤。
修复酶促过程中的小缺陷是一回事,修复大的结构性缺陷——缺陷蛋白在细胞中堆积多年——然后指望逆转损伤,则完全是另一回事。原文 (en)
Fixing small defects in enzymatic processes is one thing, fixing large structural defects, where the defect proteins pile up in cells over years, and then hoping to undo the damage is quite another.