Evidence map›Paper›PMID 30778238›Full record

ArticleNature medicine2019

Long-term evaluation of AAV-CRISPR genome editing for Duchenne muscular dystrophy.

Christopher E Nelson, Yaoying Wu, Matthew P Gemberling, Matthew L Oliver, Matthew A Waller, Joel D Bohning, Jacqueline N Robinson-Hamm, Karen Bulaklak, Ruth M Castellanos Rivera, Joel H Collier and 2 more

Abstract read
In one paragraph

Article in Nature medicine, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 291 papers.

0numbers the graph read from it
0cells of the map it votes in
291citing 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

291 citing papers in PubMed.

  1. In vivo CRISPR gene editing in patients with herpetic stromal keratitis.Molecular therapy : the journal of the American Society of Gene Therapy · 2023
    Trial
  2. CRISPR/Cas Systems: Biological Basis and Genome Editing Applications.Methods in molecular biology (Clifton, N.J.) · 2027
    Review
  3. Review
  4. Review
  5. Article
  6. Article
  7. Review
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  9. Review
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  12. In vivo systematic detection of the outcomes of CRISPR-Cas9-mediated DNA repair in skeletal muscle stem cells.Molecular therapy : the journal of the American Society of Gene Therapy · 2026
    Article
  13. SORT LNPs encapsulating Cas9 mRNA achieve efficient editing in skeletal muscle in a dystrophic mouse model.Molecular therapy : the journal of the American Society of Gene Therapy · 2026
    Article
  14. Article
  15. Review
  16. Considerations for early life genetic therapies in cystic fibrosis.American journal of physiology. Lung cellular and molecular physiology · 2026
    Review
  17. Article
  18. Review
  19. Review
  20. Review

231 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

12 authors.

Christopher E NelsonDepartment of Biomedical Engineering, Duke University, Durham, NC, USA.
Yaoying WuDepartment of Biomedical Engineering, Duke University, Durham, NC, USA.
Matthew P GemberlingDepartment of Biomedical Engineering, Duke University, Durham, NC, USA.
Matthew L OliverDepartment of Biomedical Engineering, Duke University, Durham, NC, USA.
Matthew A WallerDepartment of Biomedical Engineering, Duke University, Durham, NC, USA.
Joel D BohningDepartment of Biomedical Engineering, Duke University, Durham, NC, USA.
Jacqueline N Robinson-HammDepartment of Biomedical Engineering, Duke University, Durham, NC, USA.
Karen BulaklakDepartment of Biomedical Engineering, Duke University, Durham, NC, USA.
Ruth M Castellanos RiveraGene Therapy Center, University of North Carolina Chapel Hill, Chapel Hill, NC, USA.
Joel H CollierDepartment of Biomedical Engineering, Duke University, Durham, NC, USA.
Aravind AsokanDepartment of Surgery, Duke University Medical Center, Durham, NC, USA.
Charles A GersbachDepartment of Biomedical Engineering, Duke University, Durham, NC, USA. charles.gersbach@duke.edu.ORCID http://orcid.org/0000-0003-1478-4013

Funding

Engineering Morphogenetic Factors for Enhanced Genetic ReprogrammingDP2OD008586 · OD · DUKE UNIVERSITY · PI GERSBACH, CHARLES A. · 2011 to 2011
$2.4M
CRISPR/Cas9-Based Gene Editing for the Correction of Duchenne Muscular DystrophyR01AR069085 · NIAMS · DUKE UNIVERSITY · PI GERSBACH, CHARLES A. · 2016 to 2020
$1.7M
Non-viral delivery of CRISPR/Cas9 for targeted gene replacementK99EB023979 · NIBIB · DUKE UNIVERSITY · PI NELSON, CHRISTOPHER · 2018 to 2018
$86k
NIAMS NIH HHS R01 AR069085NIBIB NIH HHS K99 EB023979NIH HHS DP2 OD008586
6 · The paper itself

Abstract

Duchenne muscular dystrophy (DMD) is a monogenic disorder and a candidate for therapeutic genome editing. There have been several recent reports of genome editing in preclinical models of Duchenne muscular dystrophy

Indexed as

AnimalsAnimals, NewbornCRISPR-Cas SystemsDependovirusDisease Models, AnimalDystrophinGene EditingGenetic TherapyGenetic VectorsImmunity, CellularImmunity, HumoralMiceMice, Inbred mdxMuscular Dystrophy, DuchenneDystrophin

Identifiers

PMID30778238
PMCPMC6455975

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.