Prospects for prenatal gene therapy in disorders causing mental retardation

Advances in understanding the genetics and pathogenesis of disease and in prenatal diagnosis have lead to an exploration of ways to intervene earlier and earlier in the disease process. The possibility of prenatal gene therapy for severe genetic and developmental disorders has sparked new research and debate as to its feasibility, reliability, and ethics as a therapeutic option. Recent animal studies have demonstrated the feasibility of introducing a vector into the developing fetus. The optimal timing and best mode of delivery, however, have yet to be defined. Whether or not this research should be pursued also has been the subject of recent bioethical debates. There is additional concern with the possibility of in utero gene transfer inducing mutagenesis and subsequent tumor formation. This review will provide a summary of the current state of knowledge in the field of prenatal gene therapy and possible directions for the future research.