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When genes are altered so that the encoded proteins are unable to carry out their normal functions, genetic disorders can result.
Gene therapy is a technique for correcting defective genes responsible for disease development. In most gene therapy studies, a "correct copy" or "wild type" gene is inserted into the DNA of patient. A carrier called a vector must be used to deliver the therapeutic gene to the patient's target cells. Currently, the most common types of vectors are viruses that have been genetically altered to carry normal human DNA. Target cells such as the patient's liver or lung cells are infected with the vector...
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