The gene can be administered on its own or cloned into a defective virus used as a vector that can replicate but not cause infection.

对于一些疾病，骨髓被破坏，可以通过引入带有正常基因或有保护作用的基因的细胞来治疗。基因疗法虽然不成熟，但它有着巨大的潜力。