Evidence map›Paper›PMID 32687511›Full record

ArticlePloS one2020

Intranasal delivery of Thyroid hormones in MCT8 deficiency.

Carmen Grijota-Martínez, Soledad Bárez-López, Eva Ausó, Samuel Refetoff, William H Frey, Ana Guadaño-Ferraz

Open access · goldAbstract read
In one paragraph

Article in PloS one, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 4 papers.

0numbers the graph read from it
0cells of the map it votes in
4citing papers in PubMed
1.3field-weighted citation impact, top 19% 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

4 citing papers in PubMed, 12 citations in OpenAlex.

  1. Article
  2. Article
  3. Article
  4. 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

6 authors at 5 institutions in 3 countries.

Carmen Grijota-MartínezInstituto de Investigaciones Biomédicas Alberto Sols, Consejo Superior de Investigaciones Científicas (CSIC)-Universidad Autónoma de Madrid (UAM), Madrid, Spain.ORCID 0000-0003-2656-9062
Soledad Bárez-LópezCenter for Biomedical Research on Rare Diseases (Ciberer), Unit 708, Instituto de Salud Carlos III, Madrid, Spain.ORCID 0000-0002-2552-0366
Eva AusóInstituto de Investigaciones Biomédicas Alberto Sols, Consejo Superior de Investigaciones Científicas (CSIC)-Universidad Autónoma de Madrid (UAM), Madrid, Spain.
Samuel RefetoffDepartments of Medicine, The University of Chicago, Chicago, Illinois, United States of America.
William H FreyHealthPartners Neuroscience Center, St. Paul, Minnesota, United States of America.
Ana Guadaño-FerrazInstituto de Investigaciones Biomédicas Alberto Sols, Consejo Superior de Investigaciones Científicas (CSIC)-Universidad Autónoma de Madrid (UAM), Madrid, Spain.ORCID 0000-0002-0666-935X
Consejo Superior de Investigaciones Científicas · ESHealthPartners · USInstituto de Salud Carlos III · ESUniversity of Alicante · ESUniversity of Chicago · US

Funding

THYROID PHYSIOLOGY STUDIES OF INHERITED DISORDERSR01DK015070 · NIDDK · UNIVERSITY OF CHICAGO · PI ANTONIO C BIANCO, Alexandra Mihaela Dumitrescu · 1986 to 2026
$8.1M
Biotechnology and Biological Sciences Research Council BB/R016879/1NIDDK NIH HHS R01 DK015070
6 · The paper itself

Abstract

Loss of function mutations in the gene encoding the thyroid hormone transporter monocarboxylate transporter 8 (MCT8) lead to severe neurodevelopmental defects in humans associated with a specific thyroid hormone phenotype manifesting high serum 3,5,3'-triiodothyronine (T3) and low thyroxine (T4) levels. Patients present a paradoxical state of peripheral hyperthyroidism and brain hypothyroidism, this last one most likely arising from impaired thyroid hormone transport across the brain barriers. The administration of thyroid hormones by delivery pathways that bypass the brain barriers, such as the intranasal delivery route, offers the possibility to improve the neurological defects of MCT8-deficient patients. In this study, the thyroid hormones T4 and T3 were administrated intranasally in different mouse models of MCT8 deficiency. We have found that, under the present formulation, intranasal administration of thyroid hormones does not increase the content of thyroid hormones in the brain and further raises the peripheral thyroid hormone levels. Our data suggests intranasal delivery of thyroid hormones is not a suitable therapeutic strategy for MCT8 deficiency, although alternative formulations could be considered in the future to improve the nose-to-brain transport.

Indexed as

Administration, IntranasalAnimalsBrainMiceMonocarboxylic Acid TransportersMutationSignal TransductionSymportersThyroid HormonesMonocarboxylic Acid TransportersSlc16a2 protein, mouseSymportersThyroid Hormones

Identifiers

PMID32687511
PMCPMC7371167
OpenAlexW3045447744

What OpenQuestion holds

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Registered trials

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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.