Evidence map›Paper›PMID 25789154›Full record

ArticleSkeletal muscle2014

NBD delivery improves the disease phenotype of the golden retriever model of Duchenne muscular dystrophy.

Joe N Kornegay, Jennifer M Peterson, Daniel J Bogan, William Kline, Janet R Bogan, Jennifer L Dow, Zheng Fan, Jiahui Wang, Mihye Ahn, Hongtu Zhu and 2 more

Open access · goldAbstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Skeletal muscle, 2014. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 26 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
26citing papers in PubMed, 1 pooled it
1.4field-weighted citation impact, top 18% 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

26 citing papers in PubMed, 1 synthesis or guideline pooled it, 35 citations in OpenAlex.

  1. Pooled it
  2. Trial
  3. Article
  4. Article
  5. Article
  6. Emerging role of TAK1 in the regulation of skeletal muscle mass.BioEssays : news and reviews in molecular, cellular and developmental biology · 2023
    Review
  7. Article
  8. Exosome-mediated improvement in membrane integrity and muscle function in dystrophic mice.Molecular therapy : the journal of the American Society of Gene Therapy · 2021
    Article
  9. Article
  10. NF-κB inhibitor, NEMO-binding domain peptide attenuates intervertebral disc degeneration.The spine journal : official journal of the North American Spine Society · 2020
    Article
  11. The SINE Compound KPT-350 Blocks Dystrophic Pathologies in DMD Zebrafish and Mice.Molecular therapy : the journal of the American Society of Gene Therapy · 2020
    Article
  12. Review
  13. Article
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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 at 2 institutions in 1 country.

Joe N KornegayDepartment of Pathology and Laboratory Medicine, School of Medicine, University of North Carolina, Chapel Hill, NC 27599, USA ; Department of Neurology, School of Medicine, University of North Carolina, Chapel Hill, NC 27599, USA ; The Gene Therapy Center, School of Medicine, University of North Carolina, Chapel Hill, NC 27599, USA ; Department of Veterinary Integrative Biosciences, College of Veterinary Medicine, Texas A&M University, Mail Stop 4458, College Station, TX, USA.
Jennifer M PetersonDepartment of Molecular Virology, Immunology, and Medical Genetics, The Ohio State University, Columbus, OH 43210, USA.
Daniel J BoganDepartment of Pathology and Laboratory Medicine, School of Medicine, University of North Carolina, Chapel Hill, NC 27599, USA ; The Gene Therapy Center, School of Medicine, University of North Carolina, Chapel Hill, NC 27599, USA.
William KlineDepartment of Molecular Virology, Immunology, and Medical Genetics, The Ohio State University, Columbus, OH 43210, USA.
Janet R BoganDepartment of Pathology and Laboratory Medicine, School of Medicine, University of North Carolina, Chapel Hill, NC 27599, USA ; The Gene Therapy Center, School of Medicine, University of North Carolina, Chapel Hill, NC 27599, USA.
Jennifer L DowDepartment of Pathology and Laboratory Medicine, School of Medicine, University of North Carolina, Chapel Hill, NC 27599, USA ; The Gene Therapy Center, School of Medicine, University of North Carolina, Chapel Hill, NC 27599, USA.
Zheng FanDepartment of Neurology, School of Medicine, University of North Carolina, Chapel Hill, NC 27599, USA.
Jiahui WangDepartment of Psychiatry, School of Medicine, University of North Carolina, Chapel Hill, NC 27599, USA.
Mihye AhnDepartment of Biostatistics, Gillings School of Global Public Health, University of North Carolina, Chapel Hill, NC 27599, USA.
Hongtu ZhuDepartment of Biostatistics, Gillings School of Global Public Health, University of North Carolina, Chapel Hill, NC 27599, USA.
Martin StynerDepartment of Psychiatry, School of Medicine, University of North Carolina, Chapel Hill, NC 27599, USA ; Department of Computer Science, University of North Carolina, Chapel Hill, NC 27599, USA.
Denis C GuttridgeDepartment of Molecular Virology, Immunology, and Medical Genetics, The Ohio State University, Columbus, OH 43210, USA ; The Ohio State University College of Medicine, 460W. 12th Avenue, Columbus, OH 43210, USA.
University of North Carolina at Chapel Hill · USThe Ohio State University · US

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

backgroundDuchenne muscular dystrophy (DMD) is caused by mutations in the dystrophin gene and afflicts skeletal and cardiac muscles. Previous studies showed that DMD is associated with constitutive activation of NF-κB, and in dystrophin-deficient mdx and utrophin/dystrophin (utrn (-/-) ;mdx) double knock out (dko) mouse models, inhibition of NF-κB with the Nemo Binding Domain (NBD) peptide led to significant improvements in both diaphragm and cardiac muscle function.

methodsA trial in golden retriever muscular dystrophy (GRMD) canine model of DMD was initiated with four primary outcomes: skeletal muscle function, MRI of pelvic limb muscles, histopathologic features of skeletal muscles, and safety. GRMD and wild type dogs at 2 months of age were treated for 4 months with NBD by intravenous infusions. Results were compared with those collected from untreated GRMD and wild type dogs through a separate, natural history study.

resultsResults showed that intravenous delivery of NBD in GRMD dogs led to a recovery of pelvic limb muscle force and improvement of histopathologic lesions. In addition, NBD-treated GRMD dogs had normalized postural changes and a trend towards lower tissue injury on magnetic resonance imaging. Despite this phenotypic improvement, NBD administration over time led to infusion reactions and an immune response in both treated GRMD and wild type dogs.

conclusionsThis GRMD trial was beneficial both in providing evidence that NBD is efficacious in a large animal DMD model and in identifying potential safety concerns that will be informative moving forward with human trials.

Identifiers

PMID25789154
PMCPMC4364341
OpenAlexW2148202298

What OpenQuestion holds

Textmetadata
LicenceCC BY
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.