The ability to remove or alter with precision a single one ofthe thousands of genes in the body and to transmit this mutationto all subsequent progeny was a science-fiction dream only afew years ago. But today this technique is part of a routineprocedure for creating animal models that can be used to studythe pathophysiology and therapy of diseases in humans.
In general, mutations that cause a gain of function producedisease even when they occur in only one of a gene's two alleles;for example, the oncogenic mutations that cause abnormal cellproliferation. In a . . . [Full Text of this Article]
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From the Division of Endocrinology, Children's Hospital, Harvard Medical School, Boston.
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