Evidence map›Paper›PMID 31473142›Full record

ReviewTrends in molecular medicine2019

Pluripotent Stem Cell-Based Therapeutics for Muscular Dystrophies.

Sridhar Selvaraj, Michael Kyba, Rita C R Perlingeiro

Abstract readReview
In one paragraph

Review in Trends in molecular medicine, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 12 papers.

0numbers the graph read from it
0cells of the map it votes in
12citing 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

12 citing papers in PubMed.

  1. Review
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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

3 authors.

Sridhar SelvarajLillehei Heart Institute, Department of Medicine, University of Minnesota, Minneapolis, MN, USA.
Michael KybaDepartment of Pediatrics, University of Minnesota, Minneapolis, MN, USA; Stem Cell Institute, University of Minnesota, Minneapolis, MN, USA.
Rita C R PerlingeiroLillehei Heart Institute, Department of Medicine, University of Minnesota, Minneapolis, MN, USA; Stem Cell Institute, University of Minnesota, Minneapolis, MN, USA. Electronic address: perli032@umn.edu.

Funding

Probing the Activity of DUX4 in FSHDR01AR055685 · NIAMS · UNIVERSITY OF MINNESOTA · PI Michael Kyba · 2010 to 2026
$7.0M
Skeletal Muscle Regeneration from Differentiating Pluripotent Stem CellsR01AR055299 · NIAMS · UNIVERSITY OF MINNESOTA · PI PERLINGEIRO, RITA C. R. · 2008 to 2018
$3.3M
Targeting Dystroglycanopathies using Pluripotent-derived Myogenic ProgenitorsR01AR071439 · NIAMS · UNIVERSITY OF MINNESOTA · PI PERLINGEIRO, RITA C. R. · 2017 to 2021
$1.9M
NIAMS NIH HHS R01 AR055299NIAMS NIH HHS R01 AR055685NIAMS NIH HHS R01 AR071439
6 · The paper itself

Abstract

Pluripotent stem cells (PSCs) represent an attractive cell source for treating muscular dystrophies (MDs) since they easily allow for the generation of large numbers of highly regenerative myogenic progenitors. Using reprogramming technology, patient-specific PSCs have been derived for several types of MDs, and genome editing has allowed correction of mutations, opening the opportunity for their therapeutic application in an autologous transplantation setting. However, there has been limited progress on preclinical studies that validate the therapeutic potential of these gene corrected PSC-derived myogenic progenitors. In this review, we highlight the major research advances, challenges, and future prospects towards the development of PSC-based therapeutics for MDs.

Indexed as

Stem Cell TransplantationAnimalsBiomarkersCell- and Tissue-Based TherapyCell DifferentiationDisease Models, AnimalGene EditingGenetic TherapyHumansInduced Pluripotent Stem CellsMuscular DystrophiesMyoblasts, CardiacPluripotent Stem CellsRegenerationTransgenesBiomarkerscell therapygene correctionmuscular dystrophymyogenic progenitorspluripotent stem cells

Identifiers

PMID31473142
PMCPMC6721995

What OpenQuestion holds

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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.