Evidence map›Paper›PMID 32597943›Full record

ReviewJAMA pediatrics2020

Gene and Stem Cell Therapies for Fetal Care: A Review.

Amy E O'Connell, Stephanie Guseh, Larissa Lapteva, Christy L Cummings, Louise Wilkins-Haug, Jerry Chan, William H Peranteau, Graça Almeida-Porada, Stella Kourembanas

Abstract readReview
In one paragraph

Review in JAMA pediatrics, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 12 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
12citing papers in PubMed, 1 pooled it
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

12 citing papers in PubMed, 1 synthesis or guideline pooled it.

  1. Pooled it
  2. Current Status of Cellular and Gene-Based Therapies for Congenital Metabolic Disorders: A Review.Medical science monitor : international medical journal of experimental and clinical research · 2026
    Review
  3. Article
  4. Review
  5. Article
  6. Defining a Midgestational Window forbioRxiv : the preprint server for biology · 2026
    Article
  7. Review
  8. Article
  9. Article
  10. Article
  11. Review
  12. Article
4 · The record

Corrections and comments

5 · Who and what money

Authors and funding

9 authors.

Amy E O'ConnellDivision of Newborn Medicine, Boston Children's Hospital, Boston, Massachusetts.
Stephanie GusehDivision of Maternal Fetal Medicine and Reproductive Genetics, Brigham and Women's Hospital, Boston, Massachusetts.
Larissa LaptevaOffice of Tissues and Advanced Therapies, Center for Biologics Evaluation and Research, Food and Drug Administration, Silver Spring, Maryland.
Christy L CummingsDivision of Newborn Medicine, Boston Children's Hospital, Boston, Massachusetts.
Louise Wilkins-HaugDivision of Maternal Fetal Medicine and Reproductive Genetics, Brigham and Women's Hospital, Boston, Massachusetts.
Jerry ChanDepartment of Reproductive Medicine, KK Women's and Children's Hospital, Singapore.
William H PeranteauDivision of General, Thoracic and Fetal Surgery, The Children's Hospital of Philadelphia, Philadelphia, Pennsylvania.
Graça Almeida-PoradaWake Forest Institute for Regenerative Medicine, Fetal Research and Therapy Program, Winston Salem, North Carolina.
Stella KourembanasDivision of Newborn Medicine, Boston Children's Hospital, Boston, Massachusetts.

Funding

MSC Exosome Treatment for BPD: Impact on Immunity and Lung DevelopmentR01HL146128 · NHLBI · BOSTON CHILDREN'S HOSPITAL · PI Stella Kourembanas · 2019 to 2026
$6.6M
Prenatal Cell and Gene Therapy for Hemophilia AR01HL135853 · NHLBI · WAKE FOREST UNIVERSITY HEALTH SCIENCES · PI ALMEIDA-PORADA, GRACA DUARTE, PORADA, CHRISTOPHER D · 2017 to 2020
$2.8M
cGMP Manufacture Of FVIII-Expressing Placental Cells For Hemophilia AU01HL148681 · NHLBI · WAKE FOREST UNIVERSITY HEALTH SCIENCES · PI Graca Duarte Almeida-Porada · 2019 to 2026
$1.6M
NHLBI NIH HHS R01 HL135853NHLBI NIH HHS R01 HL146128NHLBI NIH HHS U01 HL148681
6 · The paper itself

Abstract

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.

Indexed as

AnimalsFemaleGenetic Diseases, InbornGenetic TherapyHumansPregnancyPrenatal CareStem Cell Transplantation

Identifiers

PMID32597943
PMCPMC10620667

What OpenQuestion holds

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LicenceTDM
Read underepoch 390

Registered trials

None linked

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.