Future AAVenues for In Utero Gene Therapy.

MacKenzie, Tippi C · Cell Stem Cell · 2018

editorial · Level V

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Abstract

Fetal gene therapy using safe and effective viral vectors no longer remains a distant prospect. Recently in Nature Medicine, Massaro et al. (2018) demonstrated that prenatal intracranial injection of a viral vector results in improved neurologic function, raising the intriguing possibility that in utero gene therapy may be approaching clinical applications.

Medical subject headings