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Dongliang Liu1, Jianjun Hu, Weihong Qiao

  • 1State Key Laboratory of Fine Chemicals, Dalian University of Technology, Dalian 116012, China. dut-liu@163.com

Bioorganic & Medicinal Chemistry Letters
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PubMed

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Gene Therapy

Gene therapy is a technique where a gene is inserted into a person’s cells to prevent or treat a serious disease. The added gene may be a healthy version of the gene that is mutated in the patient, or it could be a different gene that inactivates or compensates for the patient’s disease-causing gene. For example, in patients with severe combined immunodeficiency (SCID) due to a mutation in the gene for the enzyme adenosine deaminase, a functioning version of the gene can be inserted. The...