In utero gene therapy: prospect and future.

摘要

Many genetic disorders are reported to cause irreversible damage to the fetus before birth. In utero gene therapy may be an effective tool for correction of genetic disorders by replacing defective gene with normal one. There are many reasons for moving forward with in utero gene therapy. The most important reason is to provide early intervention as to prevent or slow dysfunction and morbidity. This approach may prove to be advantageous in rapidly replicating fetal cells, and less sensitive to immune response to vector or transgene product due to underdeveloped immune system. In addition, the developing fetus may be a better candidate for gene therapy than the adult because gene engraftment may be more feasible in early fetal life, where stem cells or pleuripotent progenitor cells are more accessible to vectors. Some reports are available on successful in utero gene transfer in animal models but many questions remain to be answered before in utero gene therapy can be considered a viable solution to human. The real moral challenge facing in utero gene therapy is finding ways to insure that the review of protocols is adequate, and that those undertaking trials are competent to do so. Present review article analyzes the overall progress of the field, and the research that still needs to be performed before it can be considered to human clinical trials.