Evidence map›Paper›PMID 31759135›Full record

ReviewNeurobiology of disease2020

Modeling cell-autonomous motor neuron phenotypes in ALS using iPSCs.

James Hawrot, Sophie Imhof, Brian J Wainger

Open access · hybridAbstract readReview
In one paragraph

Review in Neurobiology of disease, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 42 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
42citing papers in PubMed, 1 pooled it
6.1field-weighted citation impact, top 3% 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

42 citing papers in PubMed, 1 synthesis or guideline pooled it, 77 citations in OpenAlex.

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  11. Invertebrate genetic models of amyotrophic lateral sclerosis.Frontiers in molecular neuroscience · 2024
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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 at 3 institutions in 2 countries.

James HawrotDepartments of Neurology and Anesthesia, Critical Care & Pain Medicine, Massachusetts General Hospital, Boston, MA 02114, USA.
Sophie ImhofDepartments of Neurology and Anesthesia, Critical Care & Pain Medicine, Massachusetts General Hospital, Boston, MA 02114, USA; University of Amsterdam, Amsterdam, The Netherlands.
Brian J WaingerDepartments of Neurology and Anesthesia, Critical Care & Pain Medicine, Massachusetts General Hospital, Boston, MA 02114, USA; Harvard Stem Cell Institute, Cambridge, MA 02138, USA; Broad Institute of Harvard University and MIT, Cambridge, MA 02142, USA. Electronic address: brian.wainger@mgh.harvard.edu.
Harvard Stem Cell Institute · USMassachusetts General Hospital · USUniversity of Amsterdam · NL

Funding

A Human Stem Cell-Derived Neuromuscular Junction Model for Amyotrophic Lateral SclerosisDP2NS106664 · NINDS · MASSACHUSETTS GENERAL HOSPITAL · PI WAINGER, BRIAN JASON · 2017 to 2017
$2.6M
Defining an Electrophysiological Phenotype of ALS Patient-Derived Motor NeuronsK08NS082364 · NINDS · MASSACHUSETTS GENERAL HOSPITAL · PI WAINGER, BRIAN JASON · 2014 to 2018
$981k
NINDS NIH HHS DP2 NS106664NINDS NIH HHS K08 NS082364
6 · The paper itself

Abstract

Amyotrophic lateral sclerosis (ALS) is an aggressive and uniformly fatal degenerative disease of the motor nervous system. In order to understand underlying disease mechanisms, researchers leverage a host of in vivo and in vitro models, including yeast, worms, flies, zebrafish, mice, and more recently, human induced pluripotent stem cells (iPSCs) derived from ALS patients. While mouse models have been the main workhorse of preclinical ALS research, the development of iPSCs provides a new opportunity to explore molecular phenotypes of ALS within human cells. Importantly, this technology enables modeling of both familial and sporadic ALS in the relevant human genetic backgrounds, as well as a personalized or targeted approach to therapy development. Harnessing these powerful tools requires addressing numerous challenges, including different variance components associated with iPSCs and motor neurons as well as concomitant limits of reductionist approaches. In order to overcome these obstacles, optimization of protocols and assays, confirmation of phenotype robustness at scale, and validation of findings in human tissue and genetics will cement the role for iPSC models as a valuable complement to animal models in ALS and more broadly among neurodegenerative diseases.

Indexed as

Models, NeurologicalAmyotrophic Lateral SclerosisAnimalsBrainDisease Models, AnimalHumansInduced Pluripotent Stem CellsMotor NeuronsPhenotypeSpinal CordALSdisease modelgeneticsiPSCsneurodegenerative diseasetherapy

Identifiers

PMID31759135
PMCPMC7002167
OpenAlexW2991183189

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.