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基因治疗 Gene Therapy -生物名词

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The technology that uses genetic material for therapeutic purposes. This genetic material can be in the form of a gene, a representative of a gene or cDNA, RNA or even a small fragment of a gene. The introduced genetic material can be therapeutic in several ways: It can make a protein that is defective or missing in the patient’s cells (as would be the case for a genetic disorder), or one which will correct or modify a particular cellular function, or a protein that elicits an immune response. In gene therapy approaches, the genetic material may be introduced into the patient in several different ways. It can be directly injected for some applications in a process known as genetic vaccination, or it can be introduced by using bioengineered viruses that will carry the therapeutic gene as part of their own genetic cargo and deliver it into the cell. The viruses that are commonly used for this purpose are adenovirus, adeno-associated virus (AAV), retrovirus, lentivirus and herpes virus. Reagents known as liposomes can also carry therapeutic genes into cells.

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