It's a single gene defect and we know exactly where the genetic defect is in sickle cell.

是一种单基因缺陷,而且我们准确地知道,镰状红细胞贫血的基因缺陷在哪里

耶鲁公开课 - 生物医学工程探索课程节选

But it is telling that many autistic people have a genetic defect which interferes with their sulphur metabolism.

ECONOMIST: The human microbiome

Gleevec was the first cancer pill targeted at a specific genetic defect.

FORBES: Cancer Vaccine Finds A Proving Ground

These drugs were tested in small patient populations who have a specific genetic defect that the new drug was designed to repair.

FORBES: Is Big Pharma Utilizing Too Many Resources on Potential Blockbusters?