Mario Capecchi, Ph.D., Nobel Laureate

capecchi

Since nearly all biological phenomena are mediated by genes, gene targeting is impacting the analysis of nearly all aspects of mammalian biology, including studies in evolution, development, neurobiology, immunology and human disease. This technology has many applications for clinical medicine. Scientists can simulate any human genetic disease in laboratory models, study its progression, and test potential therapies against it. In the future, since the investigator can choose which gene to modify and precisely how to modify it, therapies based on gene targeting will be used to correct the endogenous defective gene in the appropriate human tissue. These therapies will be directed at the cause rather than at the symptoms of the disease.

An immediate application of gene targeting to human medicine is to generate animal models for human disease such as diabetes, atherosclerosis, hypertension and cancer. These models provide a unique opportunity to undertake in-depth analysis of the pathology of human diseases, and they offer a platform for developing new therapeutic protocols.

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