Evidence map›Paper›PMID 30193130›Full record

ArticleCell stem cell2018

Future AAVenues for In Utero Gene Therapy.

Tippi C MacKenzie

Abstract readComment
PubMed Publisher
In one paragraph

Article in Cell stem cell, 2018. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 5 papers.

0numbers the graph read from it
0cells of the map it votes in
5citing 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

5 citing papers in PubMed.

  1. Considerations for early life genetic therapies in cystic fibrosis.American journal of physiology. Lung cellular and molecular physiology · 2026
    Review
  2. Article
  3. Review
  4. Review
  5. Fetal Gene Therapy Using a Single Injection of Recombinant AAV9 Rescued SMA Phenotype in Mice.Molecular therapy : the journal of the American Society of Gene Therapy · 2019
    Article
4 · The record

Corrections and comments

5 · Who and what money

Authors and funding

1 author.

Tippi C MacKenzieThe Department of Surgery and the Center for Maternal-Fetal Precision Medicine, University of California, San Francisco, San Francisco, CA, USA. Electronic address: tippi.mackenzie@ucsf.edu.

Funding

Medical Research Council G1000709Medical Research Council MR/M00676X/1Medical Research Council MR/P026494/1Medical Research Council MR/R015325/1National Centre for the Replacement, Refinement and Reduction of Animals in Research NC/L001780/1
6 · The paper itself

Abstract

Fetal gene therapy using safe and effective viral vectors no longer remains a distant prospect. Recently in Nature Medicine, Massaro et al. (2018) demonstrated that prenatal intracranial injection of a viral vector results in improved neurologic function, raising the intriguing possibility that in utero gene therapy may be approaching clinical applications.

Indexed as

Neurodegenerative DiseasesFemaleFetusGenetic TherapyGenetic VectorsHumansInfantPregnancy

Identifiers

What OpenQuestion holds

Textmetadata
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.