用基因材料进行治疗的技术。这种基因材料可以是基因，基因替代物或cDNA、RNA甚至小的基因片段。引入的遗传材料可以在几方面有治疗作用：它可以合成一个蛋白质替代缺陷或遗失蛋白，或修正和修饰一项特定的细胞功能，或引发免疫反应。在基因治疗方法中，基因材料可以以多种方式引入病人体内。它可以以基因疫苗的方式注射，或者将携带治疗基因作为其原有基因的一部分的生物工程病毒引入体内。使用的病毒可以是腺病毒、AAV、反转录病毒、疱疹病毒。脂质体也可携带治疗基因到细胞内。

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.