Evidence map›Paper›PMID 33429080›Full record

ReviewMolecular therapy : the journal of the American Society of Gene Therapy2021

The Path to Progress Preclinical Studies of Age-Related Neurodegenerative Diseases: A Perspective on Rodent and hiPSC-Derived Models.

Gabriella MacDougall, Logan Y Brown, Boris Kantor, Ornit Chiba-Falek

Abstract readReview
In one paragraph

Review in Molecular therapy : the journal of the American Society of Gene Therapy, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 15 papers.

0numbers the graph read from it
0cells of the map it votes in
15citing 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

15 citing papers in PubMed.

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  11. Neuronal-type-specific epigenome editing to decreaseMolecular therapy. Nucleic acids · 2024
    Article
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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

4 authors.

Gabriella MacDougallDivision of Translational Brain Sciences, Department of Neurology, Duke University Medical Center, Durham, NC 27710, USA; Center for Genomic and Computational Biology, Duke University Medical Center, Durham, NC 27710, USA.
Logan Y BrownDepartment of Neurobiology, Duke University Medical Center, Durham, NC 27710, USA; Center for Advanced Genomic Technologies, Duke University Medical Center, Durham, NC 27710, USA; Viral Vector Core, Duke University Medical Center, Durham, NC 27710, USA.
Boris KantorDepartment of Neurobiology, Duke University Medical Center, Durham, NC 27710, USA; Center for Advanced Genomic Technologies, Duke University Medical Center, Durham, NC 27710, USA; Viral Vector Core, Duke University Medical Center, Durham, NC 27710, USA. Electronic address: boris.kantor@duke.edu.
Ornit Chiba-FalekDivision of Translational Brain Sciences, Department of Neurology, Duke University Medical Center, Durham, NC 27710, USA; Center for Genomic and Computational Biology, Duke University Medical Center, Durham, NC 27710, USA. Electronic address: o.chibafalek@duke.edu.

Funding

Lewy body neuropathologies and SNCA gene: variants expression and splicingRF1NS113548 · NINDS · DUKE UNIVERSITY · PI CHIBA-FALEK, ORNIT · 2020 to 2020
$3.8M
Deciphering the regulation of gene expression in the etiology of LOADR01AG057522 · NIA · DUKE UNIVERSITY · PI CHIBA-FALEK, ORNIT, LUTZ, MICHAEL WILLIAM · 2017 to 2021
$3.6M
NIA NIH HHS R01 AG057522NINDS NIH HHS RF1 NS113548
6 · The paper itself

Abstract

Alzheimer's disease (AD) and Parkinson's disease (PD) are the two most prevalent age-related neurodegenerative diseases, and currently no effective clinical treatments exist for either, despite decades of clinical trials. The failure to translate preclinical findings into effective treatments is indicative of a problem in the current evaluation pipeline for potential therapeutics. At present, there are no useful animal models for AD and PD research that reflect the entire biology of the diseases, specifically, the more common non-Mendelian forms. Whereas the field continues to seek suitable rodent models for investigating potential therapeutics for these diseases, rodent models have still been used primarily for preclinical studies. Here, we advocate for a paradigm shift toward the application of human-induced pluripotent stem cell (hiPSC)-derived systems for PD and AD modeling and the development of improved human-based models in a dish for drug discovery and preclinical assessment of therapeutic targets.

Indexed as

Disease Models, AnimalAlzheimer DiseaseAnimalsHumansInduced Pluripotent Stem CellsParkinson DiseaseRodentiaStem Cell Transplantation

Identifiers

PMID33429080
PMCPMC7934639

What OpenQuestion holds

Textmetadata
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.