Evidence map›Paper›PMID 33037407›Full record

ReviewGene therapy2022

Gene therapy and gene correction: targets, progress, and challenges for treating human diseases.

Matthew R Cring, Val C Sheffield

Open access · bronzeAbstract readReview
PubMed Publisher
In one paragraph

Review in Gene therapy, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 63 papers.

0numbers the graph read from it
0cells of the map it votes in
63citing papers in PubMed
4.9field-weighted citation impact, top 4% of its field
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

63 citing papers in PubMed, 149 citations in OpenAlex.

  1. Article
  2. Review
  3. Optimization of CEST MRI Reporter Protein Design Using Cation-Pi Networks.Chemistry (Weinheim an der Bergstrasse, Germany) · 2026
    Article
  4. Review
  5. Review
  6. Review
  7. Review
  8. Article
  9. Review
  10. Gene Therapy for Heart Failure.Journal of cardiovascular translational research · 2026
    Review
  11. Review
  12. Review
  13. Review
  14. Article
  15. Article
  16. Review
  17. Review
  18. Article
  19. Article
  20. Review

3 more citing papers are in PubMed but not listed here.

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

2 authors at 1 institution in 1 country.

Matthew R CringDepartment of Pediatrics, Division of Medical Genetics and Genomics, University of Iowa, Iowa City, IA, USA.
Val C SheffieldDepartment of Pediatrics, Division of Medical Genetics and Genomics, University of Iowa, Iowa City, IA, USA. val-sheffield@uiowa.edu.ORCID http://orcid.org/0000-0002-6282-0835
University of Iowa · US

Funding

Treatment of a Complex Retinal Degenerative SyndromeR01EY011298 · NEI · UNIVERSITY OF IOWA · PI SHEFFIELD, VAL C. · 1996 to 2017
$7.3M
Stem Cell and Organoid CoreP30EY025580 · NEI · UNIVERSITY OF IOWA · PI Budd A Tucker · 2016 to 2026
$6.9M
Interdisciplinary approach to retinal disease gene identificationR01EY017168 · NEI · UNIVERSITY OF IOWA · PI SHEFFIELD, VAL C. · 2007 to 2017
$5.9M
NEI NIH HHS P30 EY025580NEI NIH HHS R01 EY011298NEI NIH HHS R01 EY017168
6 · The paper itself

Abstract

The field of gene therapy has made significant strides over the last several decades toward the treatment of previously untreatable genetic disease. Gene therapy techniques have been aimed at mitigating disease features of recessive and dominant disorders, as well as several cancers and other diseases. While there have been numerous disease targets of gene therapy trials, only four therapies have reached FDA and/or EMA approval for clinical use. Gene correction using CRISPR-Cas9 is an extension of gene therapy that has received considerable attention in recent years and boasts many possible uses beyond classical gene therapy approaches. While there is significant therapeutic potential using gene therapy and gene correction strategies, a number of hurdles remain to be overcome before they become more common in clinical use, particularly with regards to safety and efficacy. As research progresses in this exciting field, it is likely that these therapies will become first-line treatments and will have tremendous positive impacts on the lives of patients with genetic disorders.

Indexed as

Gene EditingNeoplasmsCRISPR-Cas SystemsGenetic TherapyHumans

Identifiers

PMID33037407
OpenAlexW3092176100

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.