Evidence map›Paper›PMID 33370574›Full record

ReviewNeuroscience and biobehavioral reviews2021

Patient-derived iPSC modeling of rare neurodevelopmental disorders: Molecular pathophysiology and prospective therapies.

K R Sabitha, Ashok K Shetty, Dinesh Upadhya

Open access · greenAbstract readReview
In one paragraph

Review in Neuroscience and biobehavioral reviews, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 30 papers, 2 of them syntheses that pooled it.

0numbers the graph read from it
0cells of the map it votes in
30citing papers in PubMed, 2 pooled it
5.0field-weighted citation impact, top 4% 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

30 citing papers in PubMed, 2 syntheses or guidelines pooled it, 48 citations in OpenAlex.

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  14. [Glycosylphosphatidylinositol biosynthesis deficiency 15 caused byZhongguo dang dai er ke za zhi = Chinese journal of contemporary pediatrics · 2023
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  19. Editorial:Frontiers in neuroscience · 2023
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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 2 institutions in 2 countries.

K R SabithaCentre for Molecular Neurosciences, Kasturba Medical College, Manipal Academy of Higher Education, Manipal, 576104, Karnataka, India.
Ashok K ShettyInstitute for Regenerative Medicine, Department of Molecular and Cellular Medicine, Texas A&M University College of Medicine, College Station, TX, USA. Electronic address: akskrs@tamu.edu.
Dinesh UpadhyaCentre for Molecular Neurosciences, Kasturba Medical College, Manipal Academy of Higher Education, Manipal, 576104, Karnataka, India. Electronic address: dinesh.upadhya@manipal.edu.
Manipal Academy of Higher Education · INTexas A&M University · US

Funding

Mesenchymal Stem Cell Derived Extracellular Vesicles for Traumatic Brain InjuryR01NS106907 · NINDS · TEXAS A&M UNIVERSITY HEALTH SCIENCE CTR · PI ASHOK K SHETTY · 2018 to 2026
$4.0M
NINDS NIH HHS R01 NS106907
6 · The paper itself

Abstract

The pathological alterations that manifest during the early embryonic development due to inherited and acquired factors trigger various neurodevelopmental disorders (NDDs). Besides major NDDs, there are several rare NDDs, exhibiting specific characteristics and varying levels of severity triggered due to genetic and epigenetic anomalies. The rarity of subjects, paucity of neural tissues for detailed analysis, and the unavailability of disease-specific animal models have hampered detailed comprehension of rare NDDs, imposing heightened challenge to the medical and scientific community until a decade ago. The generation of functional neurons and glia through directed differentiation protocols for patient-derived iPSCs, CRISPR/Cas9 technology, and 3D brain organoid models have provided an excellent opportunity and vibrant resource for decoding the etiology of brain development for rare NDDs caused due to monogenic as well as polygenic disorders. The present review identifies cellular and molecular phenotypes demonstrated from patient-derived iPSCs and possible therapeutic opportunities identified for these disorders. New insights to reinforce the existing knowledge of the pathophysiology of these disorders and prospective therapeutic applications are discussed.

Indexed as

Induced Pluripotent Stem CellsNeurodevelopmental DisordersAnimalsCell DifferentiationHumansNeuronsProspective Studies3D brain organoidsAlexander diseaseAngelman syndromeCRISR/Cas9 technologyDirected differentiationDravet syndromeFragile X syndromeFriedreich’s ataxiaHuntington’s diseaseInduced pluripotent stem cellsMiller dieker syndromePhelan-McDermid syndromeRare neurodevelopmental disordersRett syndromeSpinal muscular atrophy

Identifiers

PMID33370574
PMCPMC7962756
OpenAlexW3113445861

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.