Evidence map›Paper›PMID 31706840›Full record

ArticleTrends in molecular medicine2020

Challenges and Opportunities for Translation of Therapies to Improve Cognition in Down Syndrome.

Sarah E Lee, Monica Duran-Martinez, Sabina Khantsis, Diana W Bianchi, Faycal Guedj

Open access · greenAbstract read
In one paragraph

Article in Trends in molecular medicine, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 16 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
16citing papers in PubMed, 1 pooled it
2.7field-weighted citation impact, top 10% 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

16 citing papers in PubMed, 1 synthesis or guideline pooled it, 23 citations in OpenAlex.

  1. Pooled it
  2. Article
  3. Neuronal oscillations in cognition: Down syndrome as a model of mouse to human translation.The Neuroscientist : a review journal bringing neurobiology, neurology and psychiatry · 2025
    Review
  4. Article
  5. Review
  6. The Role of Oxidative Stress in Trisomy 21 Phenotype.Cellular and molecular neurobiology · 2023
    Review
  7. Psychometric properties of inhibitory control measures among youth with Down syndrome.Journal of intellectual disability research : JIDR · 2023
    Article
  8. Article
  9. Article
  10. Article
  11. Review
  12. Article
  13. Article
  14. Review
  15. Article
  16. Review
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

5 authors at 1 institution in 1 country.

Sarah E LeeMedical Genetics Branch (Prenatal Genomic and Therapy Section), National Human Genome Research Institute, National Institutes of Health, Bethesda, MD 20892, USA. Electronic address: sarah.lee2@nih.gov.
Monica Duran-MartinezMedical Genetics Branch (Prenatal Genomic and Therapy Section), National Human Genome Research Institute, National Institutes of Health, Bethesda, MD 20892, USA.
Sabina KhantsisMedical Genetics Branch (Prenatal Genomic and Therapy Section), National Human Genome Research Institute, National Institutes of Health, Bethesda, MD 20892, USA.
Diana W BianchiMedical Genetics Branch (Prenatal Genomic and Therapy Section), National Human Genome Research Institute, National Institutes of Health, Bethesda, MD 20892, USA; Eunice Kennedy Shriver National Institute of Child Health and Human Development, National Institutes of Health, Bethesda 20892, MD, USA.
Faycal GuedjMedical Genetics Branch (Prenatal Genomic and Therapy Section), National Human Genome Research Institute, National Institutes of Health, Bethesda, MD 20892, USA. Electronic address: faycal.guedj@nih.gov.
National Institutes of Health · US

Funding

Prenatal Treatment of Down Syndrome to Improve Brain Development and NeurocognitionZIAHG200399 · NHGRI · NATIONAL HUMAN GENOME RESEARCH INSTITUTE · PI BIANCHI, DIANA · 2017 to 2025
$11.1M
Intramural NIH HHS ZIA HG200399
6 · The paper itself

Abstract

While preclinical studies have reported improvement of behavioral deficits in the Ts65Dn mouse model of Down syndrome (DS), translation to human clinical trials to improve cognition in individuals with DS has had a poor success record. Timing of the intervention, choice of animal models, strategy for drug selection, and lack of translational endpoints between animals and humans contributed to prior failures of human clinical trials. Here, we focus on in vitro cell models from humans with DS to identify the molecular mechanisms underlying the brain phenotype associated with DS. We emphasize the importance of using these cell models to screen for therapeutic molecules, followed by validating them in the most suitable animal models prior to initiating human clinical trials.

Indexed as

AnimalsBrainChromosomes, Human, Pair 21CognitionCognition DisordersDisease Models, AnimalDown SyndromeHumansPhenotypePluripotent Stem Cells

Identifiers

PMID31706840
PMCPMC6997046
OpenAlexW2982935171

What Socratic 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 Socratic graph.