Evidence map›Paper›PMID 30609814›Full record

ReviewCells2019

iPSCs as a Platform for Disease Modeling, Drug Screening, and Personalized Therapy in Muscular Dystrophies.

Jose L Ortiz-Vitali, Radbod Darabi

Open access · goldAbstract readReview
In one paragraph

Review in Cells, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 26 papers.

0numbers the graph read from it
0cells of the map it votes in
26citing papers in PubMed
2.9field-weighted citation impact, top 8% 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, 49 citations in OpenAlex.

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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

2 authors at 1 institution in 1 country.

Jose L Ortiz-VitaliCenter for Stem Cell and Regenerative Medicine (CSCRM), The Brown Foundation Institute of Molecular Medicine for the Prevention of Human Diseases (IMM), The University of Texas Health Science Center at Houston, Houston, TX 77030, USA. Jose.L.OrtizVitali@uth.tmc.edu.ORCID 0000-0002-6326-1529
Radbod DarabiCenter for Stem Cell and Regenerative Medicine (CSCRM), The Brown Foundation Institute of Molecular Medicine for the Prevention of Human Diseases (IMM), The University of Texas Health Science Center at Houston, Houston, TX 77030, USA. Radbod.Darabi@uth.tmc.edu.
Brown Foundation · US

Funding

Study of Skeletal Muscle Differentiation in Human iPS Cells by Knock-in ReportersR01AR068293 · NIAMS · UNIVERSITY OF TEXAS HLTH SCI CTR HOUSTON · PI DARABI, RADBOD · 2016 to 2020
$1.7M
Identification of the Novel Regulatory Pathways in Skeletal Myogenesis Using a Genome-Scale Lentiviral sgRNA Library ScreenR21AR071583 · NIAMS · UNIVERSITY OF TEXAS HLTH SCI CTR HOUSTON · PI DARABI, RADBOD · 2017 to 2018
$373k
NIAMS NIH HHS R01 AR068293NIAMS NIH HHS R21 AR071583
6 · The paper itself

Abstract

Induced pluripotent stem cells (iPSCs) are the foundation of modern stem cell-based regenerative medicine, especially in the case of degenerative disorders, such as muscular dystrophies (MDs). Since their introduction in 2006, many studies have used iPSCs for disease modeling and identification of involved mechanisms, drug screening, as well as gene correction studies. In the case of muscular dystrophies, these studies commenced in 2008 and continue to address important issues, such as defining the main pathologic mechanisms in different types of MDs, drug screening to improve skeletal/cardiac muscle cell survival and to slow down disease progression, and evaluation of the efficiency of different gene correction approaches, such as exon skipping, Transcription activator-like effector nucleases (TALENs), Zinc finger nucleases (ZFNs) and RNA-guided endonuclease Cas9 (CRISPR/Cas9). In the current short review, we have summarized chronological progress of these studies and their key findings along with a perspective on the future road to successful iPSC-based cell therapy for MDs and the potential hurdles in this field.

Indexed as

Precision MedicineAnimalsCRISPR-Cas SystemsDrug Evaluation, PreclinicalGene TargetingHumansInduced Pluripotent Stem CellsModels, BiologicalMuscular Dystrophy, DuchenneTranscription Activator-Like Effector NucleasesZinc Finger NucleasesTranscription Activator-Like Effector NucleasesZinc Finger NucleasesCRISPR/Cas9DMDduchennegene correctioniPSCsmuscular dystrophiesstem cells

Identifiers

PMID30609814
PMCPMC6356384
OpenAlexW2906991869

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.