InUtero Gene Therapy: Progress and Challenges

Trends Mol Med. 2021 Aug;27(8):728-730. doi: 10.1016/j.molmed.2021.05.007. Epub 2021 Jun 24.

Abstract

In utero gene therapy has the potential to treat lethal and morbid perinatal diseases before birth. Small fetal size, a tolerogenic immune system, and dosing efficiency make the fetus a compelling patient. Numerous clinical, social, and institutional factors must be considered to achieve the promise of genetic treatment before birth.

Keywords: CRISPR; fetal; gene editing; gene therapy; in utero; lipid nanoparticle.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't
  • Review

MeSH terms

  • CRISPR-Cas Systems
  • Embryonic Development / genetics*
  • Female
  • Fetus / metabolism*
  • Gene Editing
  • Gene Transfer Techniques*
  • Genetic Therapy / methods*
  • Humans
  • Nanotechnology
  • Pregnancy
  • Theranostic Nanomedicine
  • Translational Research, Biomedical