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June 29, 2020

Gene and Stem Cell Therapies for Fetal Care: A Review

Author Affiliations
  • 1Division of Newborn Medicine, Boston Children’s Hospital, Boston, Massachusetts
  • 2Department of Pediatrics, Harvard Medical School, Boston, Massachusetts
  • 3Division of Maternal Fetal Medicine and Reproductive Genetics, Brigham and Women’s Hospital, Boston, Massachusetts
  • 4Office of Tissues and Advanced Therapies, Center for Biologics Evaluation and Research, Food and Drug Administration, Silver Spring, Maryland
  • 5Department of Reproductive Medicine, KK Women’s and Children’s Hospital, Singapore
  • 6Duke-NUS Medical School, Academic Program in Obstetrics and Gynaecology, Singapore
  • 7Division of General, Thoracic and Fetal Surgery, The Children’s Hospital of Philadelphia, Philadelphia, Pennsylvania
  • 8Wake Forest Institute for Regenerative Medicine, Fetal Research and Therapy Program, Winston Salem, North Carolina
JAMA Pediatr. Published online June 29, 2020. doi:10.1001/jamapediatrics.2020.1519

Importance  Gene and stem cell therapies have become viable therapeutic options for many postnatal disorders. For select conditions, prenatal application would provide improved outcomes. The fetal state allows for several theoretical advantages over postnatal therapy, including immune immaturity and cellular niche accessibility.

Observations  Advances in prenatal diagnostic accuracy and surgical precision, as well as improvements in stem cell and gene therapy methods, have made prenatal gene and stem cell therapy realistic. Studies in mouse models and early human trials demonstrate the feasibility of these approaches. Additional efforts are under way to streamline fetal applications of stem cell and gene therapy while carefully considering best ethical practice and following established regulatory pathways.

Conclusions and Relevance  Fetal stem cell and gene therapy bring important therapeutic opportunities for select disorders that present in the fetal and neonatal periods. While this field is in its infancy, these therapies are starting to be available clinically, and clinicians should be aware of their benefits and challenges.

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