The present and future role of gene therapy in the newborn

There are currently a large number of ongoing gene therapy trials in North America and Europe. These trials have almost exclusively involved patients with inherited lethal disorders or malignancies. Although there are significant ethical and safety issues that remain unresolved, it seems inevitable that this technology will soon be adapted for use in lethal, or potentially lethal, fetal and neonatal diseases. If ethical and safety issues can be resolved, a wide spectrum of nonlethal acute and chronic diseases could also benefit from this form of therapy. The purpose of this brief review is to provide an overview of current approaches to gene delivery, their successes, and their limitations. Where possible, the discussion has focused on conditions that are recognized in fetal or neonatal life, to give the reader some sense of the potential scope for this form of therapy.