Haploinsufficiency --- A cause of genetic disease in which the contribution from a normal allele is insufficient to prevent disease because of a loss-of-functionmutation at the other allele.
遗传病的原因之一,单倍的基因剂量不足以维持正常功能。
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Unlike a genetic mutation that changes the function or activity of a gene, which can be inhibited by a well-tailored drug, loss of a gene leaves nothing for the drug to target.