Evidence map›Paper›PMID 26454198›Full record

ReviewMethods (San Diego, Calif.)2016

Gene therapy in monogenic congenital myopathies.

Xuan Guan, Melissa A Goddard, David L Mack, Martin K Childers

Open access · greenAbstract readReview
PubMed Full text at PMC Publisher
In one paragraph

Review in Methods (San Diego, Calif.), 2016. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 3 papers.

0numbers the graph read from it
0cells of the map it votes in
3citing papers in PubMed
1.1field-weighted citation impact, top 24% 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

3 citing papers in PubMed, 11 citations in OpenAlex.

  1. Article
  2. Review
  3. Systemic AAV8-Mediated Gene Therapy Drives Whole-Body Correction of Myotubular Myopathy in Dogs.Molecular therapy : the journal of the American Society of Gene Therapy · 2017
    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

4 authors at 2 institutions in 1 country.

Xuan GuanDepartment of Physiology and Pharmacology, School of Medicine, Wake Forest University Health Sciences, Winston-Salem, NC, USA; Department of Rehabilitation Medicine, University of Washington, Seattle, WA, USA; Institute for Stem Cell and Regenerative Medicine, University of Washington, Seattle, WA, USA.
Melissa A GoddardDepartment of Physiology and Pharmacology, School of Medicine, Wake Forest University Health Sciences, Winston-Salem, NC, USA; Department of Rehabilitation Medicine, University of Washington, Seattle, WA, USA; Institute for Stem Cell and Regenerative Medicine, University of Washington, Seattle, WA, USA.
David L MackDepartment of Rehabilitation Medicine, University of Washington, Seattle, WA, USA; Institute for Stem Cell and Regenerative Medicine, University of Washington, Seattle, WA, USA.
Martin K ChildersDepartment of Rehabilitation Medicine, University of Washington, Seattle, WA, USA; Institute for Stem Cell and Regenerative Medicine, University of Washington, Seattle, WA, USA. Electronic address: mkc8@uw.edu.
University of Washington · USWake Forest University · US

Funding

Gene therapy in canine myotubular myopathy for clinical translationR01HL115001 · NHLBI · UNIVERSITY OF WASHINGTON · PI CHILDERS, MARTIN K · 2013 to 2017
$3.9M
Establishing endpoints in canine myotubular myopathy for clinical translationR21AR064503 · NIAMS · UNIVERSITY OF WASHINGTON · PI CHILDERS, MARTIN K · 2012 to 2013
$389k
NHLBI NIH HHS R01 HL115001NIAMS NIH HHS R21 AR064503
6 · The paper itself

Abstract

Current treatment options for patients with monogenetic congenital myopathies (MCM) ameliorate the symptoms of the disorder without resolving the underlying cause. However, gene therapies are being developed where the mutated or deficient gene target is replaced. Preclinical findings in animal models appear promising, as illustrated by gene replacement for X-linked myotubular myopathy (XLMTM) in canine and murine models. Prospective applications and approaches to gene replacement therapy, using these disorders as examples, are discussed in this review.

Indexed as

Genetic TherapyAnimalsGene Transfer TechniquesHumansMuscle ProteinsMyopathies, Structural, CongenitalMuscle ProteinsAnimal modelsGene therapyMuscle diseaseMuscular dystrophyMyopathy

Identifiers

PMID26454198
PMCPMC4826856
OpenAlexW1931434920

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.