In utero gene editing and precision medicine

Advances in preconception and prenatal genetic testing, in combination with the rapid evolution and regulatory acceptance of targeted gene therapy technologies, has unveiled promising opportunities for the antenatal treatment of genetic disease by way of in utero gene editing and precision medicine. Recent regulatory guidance, put forth by the Federal Drug Administration (FDA), on gene editing has the exciting potential to accelerate the development and implementation of therapeutics for rare and ultrarare diseases. In this review, we highlight recent advances that have occurred in the fields of in utero gene editing and precision medicine and the opportune regulatory landscape that may facilitate the clinical translation of therapeutics in the future. We additionally review the indispensable role of prenatal genetic diagnosis, including chorionic villus sampling, amniocentesis, and noninvasive prenatal testing, as the diagnostic gateway through which candidates for in utero gene and precision therapies will be identified.