In Utero Gene Therapy and Genome Editing

@article{Hartman2018InUG,
  title={In Utero Gene Therapy and Genome Editing},
  author={Heather A. Hartman and Avery C. Rossidis and William H. Peranteau},
  journal={Current Stem Cell Reports},
  year={2018},
  pages={1-9}
}
The purpose of this review is to summarize the current status of in utero gene therapy and postnatal in vivo genome editing with an eye toward prenatal genome editing. The rational for gene therapy and genome editing in utero is described, specifically the small size of the fetus, fetal immunologic immaturity, prenatal accessibility of stem and progenitor cells, and the ability to treat target diseases prior to birth. We review studies in normal and disease small and large animal models which… CONTINUE READING