Evidence map›Paper›PMID 34176774›Full record

ReviewTrends in molecular medicine2021

InUtero Gene Therapy: Progress and Challenges.

Sourav K Bose, Pallavi Menon, William H Peranteau

Abstract readReview
In one paragraph

Review in Trends in molecular medicine, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 13 papers.

0numbers the graph read from it
0cells of the map it votes in
13citing papers in PubMed
–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

13 citing papers in PubMed.

  1. Article
  2. Review
  3. Article
  4. Review
  5. Article
  6. Non-Viral RNA Delivery During Pregnancy: Opportunities and Challenges.Small (Weinheim an der Bergstrasse, Germany) · 2024
    Review
  7. In utero delivery of targeted ionizable lipid nanoparticles facilitates in vivo gene editing of hematopoietic stem cells.Proceedings of the National Academy of Sciences of the United States of America · 2024
    Article
  8. Article
  9. Article
  10. Article
  11. Article
  12. Review
  13. Review
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

3 authors.

Sourav K BoseThe Center for Fetal Research, Division of General, Thoracic and Fetal Surgery, The Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA.
Pallavi MenonThe Center for Fetal Research, Division of General, Thoracic and Fetal Surgery, The Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA.
William H PeranteauThe Center for Fetal Research, Division of General, Thoracic and Fetal Surgery, The Children's Hospital of Philadelphia, Philadelphia, PA 19104, USA. Electronic address: peranteauw@email.chop.edu.

Funding

In utero gene editing to cure a metabolic liver diseaseR01DK123049 · NIDDK · CHILDREN'S HOSP OF PHILADELPHIA · PI PERANTEAU, WILLIAM H. · 2020 to 2024
$3.7M
In utero CRISPR-mediated gene editing to cure congenital monogenic disordersDP2HL152427 · NHLBI · CHILDREN'S HOSP OF PHILADELPHIA · PI PERANTEAU, WILLIAM H. · 2019 to 2019
$2.6M
Prenatal pulmonary cell gene editing to cure monogenic lung diseasesR01HL151352 · NHLBI · CHILDREN'S HOSP OF PHILADELPHIA · PI PERANTEAU, WILLIAM H. · 2020 to 2022
$1.2M
NHLBI NIH HHS DP2 HL152427NHLBI NIH HHS R01 HL151352NIDDK NIH HHS R01 DK123049
6 · The paper itself

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.

Indexed as

Gene Transfer TechniquesCRISPR-Cas SystemsEmbryonic DevelopmentFemaleFetusGene EditingGenetic TherapyHumansNanotechnologyPregnancyTheranostic NanomedicineTranslational Research, BiomedicalCRISPRfetalgene editinggene therapyin uterolipid nanoparticle

Identifiers

PMID34176774
PMCPMC8713252

What OpenQuestion holds

Textmetadata
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.