Organ targeted prenatal gene therapy-how far are we?

Prenatal gene therapy aims to deliver genes to cells and tissues early in prenatal life, allowing correction of a genetic defect, before long‐term tissue damage has occurred. In contrast to postnatal gene therapy, prenatal application can target genes to a large population of dividing stem cells, and the smaller fetal size allows a higher vector‐to‐target cell ratio to be achieved. Early‐gestation delivery may allow the development of immune...