Evidence map›Paper›PMID 23553671›Full record

ReviewMuscle & nerve2013

Gene and cell-mediated therapies for muscular dystrophy.

Patryk Konieczny, Kristy Swiderski, Jeffrey S Chamberlain

Open access · greenAbstract readReview
In one paragraph

Review in Muscle & nerve, 2013. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 52 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
52citing papers in PubMed, 1 pooled it
5.1field-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

52 citing papers in PubMed, 1 synthesis or guideline pooled it, 78 citations in OpenAlex.

  1. Pooled it
  2. Article
  3. Article
  4. PET reporter systems for the brain.Trends in neurosciences · 2023
    Review
  5. Article
  6. CORP: Gene delivery into murine skeletal muscle using in vivo electroporation.Journal of applied physiology (Bethesda, Md. : 1985) · 2022
    Review
  7. Article
  8. Duchenne Muscular Dystrophy: the Heart of the Matter.Current heart failure reports · 2020
    Review
  9. Article
  10. Article
  11. Article
  12. Review
  13. Article
  14. Review
  15. Article
  16. Review
  17. Article
  18. Review
  19. Article
  20. Article
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 at 1 institution in 1 country.

Patryk KoniecznyDepartment of Neurology, The University of Washington School of Medicine, Seattle, Washington 98105, USA.
Kristy Swiderski
Jeffrey S Chamberlain
University of Washington · US

Funding

Dystrophin Replacement in mdx miceR37AR040864 · NIAMS · UNIVERSITY OF WASHINGTON · PI CHAMBERLAIN, JEFFREY S · 2005 to 2014
$5.0M
Genetic modification of aging and diseased striated muscleR01AG033610 · NIA · UNIVERSITY OF WASHINGTON · PI CHAMBERLAIN, JEFFREY S · 2009 to 2013
$1.9M
NIAMS NIH HHS R37 AR040864NIA NIH HHS R01 AG033610
6 · The paper itself

Abstract

Duchenne muscular dystrophy (DMD) is a devastating muscle disorder that affects 1 in 3,500 boys. Despite years of research and considerable progress in understanding the molecular mechanism of the disease and advancement of therapeutic approaches, there is no cure for DMD. The current treatment options are limited to physiotherapy and corticosteroids, and although they provide a substantial improvement in affected children, they only slow the course of the disorder. On a more optimistic note, more recent approaches either significantly alleviate or eliminate muscular dystrophy in murine and canine models of DMD and importantly, many of them are being tested in early phase human clinical trials. This review summarizes advancements that have been made in viral and nonviral gene therapy as well as stem cell therapy for DMD with a focus on the replacement and repair of the affected dystrophin gene.

Indexed as

Genetic TherapyStem Cell TransplantationHumansMuscular Dystrophies

Identifiers

PMID23553671
PMCPMC4077844
OpenAlexW2143203978

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.