Prospects for prenatal gene therapy in disorders causing mental retardation

Advances in understanding the genetics and pathogenesis of disease and in p renatal diagnosis have lead to an exploration of ways to intervene earlier and earlier in the disease process. The possibility of prenatal gene therap y for severe genetic and developmental disorders has sparked new research a nd debate as to its feasibility, reliability, and ethics as a therapeutic o ption. Recent animal studies have demonstrated the feasibility of introduci ng 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 sho uld be pursued also has been the subject of recent bioethical debates. Ther e is additional concern with the possibility of in utero gene transfer indu cing mutagenesis and subsequent tumor formation. This review will provide a summary of the current state of knowledge in the field of prenatal gene th erapy and possible directions for the future research. (C) 2001 Wiley-Liss, Inc.