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U.S. Department of Health and Human Services

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JAMA Pediatr 2020 Jun 29 [Epub ahead of print]

Gene and stem cell therapies for fetal care: a review.

O'Connell AE, Guseh S, Lapteva L, Cummings CL, Wilkins-Haug L, Chan J, Peranteau WH, Almeida-Porada G, Kourembanas S


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.

Category: Journal Article, Review
PubMed ID: #32597943 DOI: 10.1001/jamapediatrics.2020.1519
Includes FDA Authors from Scientific Area(s): Biologics
Entry Created: 2020-07-05 Entry Last Modified: 2020-07-12