Opportunities and Challenges of Fetal Gene Therapy

Fetal gene therapy represents a promising approach to treat severe congenital disorders by intervening during a critical developmental period. This review aims to explore the unique advantages, specific applications, and challenges of this early intervention strategy.

We reviewed preclinical and emerging clinical studies evaluating the biological rationale, feasibility, and therapeutic potential of gene therapy administered in utero.

The fetal window offers unique advantages, including enhanced tissue accessibility and biodistribution, immune tolerance to new therapeutic molecules, and has the potential to prevent irreversible organ damage before birth. However, this approach requires ethical considerations including risks to both the fetus and mother, complexities of informed consent, and broad societal implications.

Although substantial challenges remain, fetal gene therapy has the potential to improve outcomes in early-onset severe disorders that currently lack effective postnatal treatments.