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Intra‐amniotic viral gene transfer highlights the potential for in utero molecular therapies

The emergence of the field of fetal somatic gene therapy is becoming increasingly recognized as an alternative and viable approach for ameliorating the burden and irreversible manifestations of many inherited conditions. The in utero administration of a therapeutic gene may allow targeting of expanding stem cell populations of organ or cell systems inaccessible later in life and, furthermore, help to avoid immune sensitization against the therapeutic vector system or transgene protein product. In addition, the progress in development of detailed fetal anatomical profiling and interventional techniques has rapidly advanced the means to administer a therapeutic gene transfer vector to the fetus in utero. The potential for fetal gene therapy to offer a cure for certain genetic diseases would introduce another much needed management option following prenatal diagnosis of inherited disease where termination or acceptance of an affected child are presently the only options available. The growing interest in the field of fetal gene therapy is highlighted most recently by at least three publications that address important considerations in the selection of appropriate candidate diseases, routes of administration, applicability of current vector systems, timing in fetal development for therapeutic gene transfer and efficacy of expression and outcome in relevant animal models.