Evidence map›Paper›PMID 33102615›Full record

ArticleMolecular therapy. Methods & clinical development2020

Pilar Muñoz, María Tristán-Manzano, Almudena Sánchez-Gilabert, Giorgia Santilli, Anne Galy, Adrian J Thrasher, Francisco Martin

Open access · goldAbstract read
In one paragraph

Article in Molecular therapy. Methods & clinical development, 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
0.7field-weighted citation impact, top 25% 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, 6 citations in OpenAlex.

  1. Review
  2. Article
  3. Article
  4. Role of Thrombopoietin Receptor Agonists in Inherited Thrombocytopenia.International journal of molecular sciences · 2021
    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

7 authors at 3 institutions in 3 countries.

Pilar MuñozGenomic Medicine Department, GENYO, Centre for Genomics and Oncological Research, Pfizer-University of Granada-Andalusian Regional Government, Parque Tecnológico Ciencias de la Salud (PTS), Avenida de la Ilustracion 114, 18016 Granada, Spain.
María Tristán-ManzanoGenomic Medicine Department, GENYO, Centre for Genomics and Oncological Research, Pfizer-University of Granada-Andalusian Regional Government, Parque Tecnológico Ciencias de la Salud (PTS), Avenida de la Ilustracion 114, 18016 Granada, Spain.
Almudena Sánchez-GilabertGenomic Medicine Department, GENYO, Centre for Genomics and Oncological Research, Pfizer-University of Granada-Andalusian Regional Government, Parque Tecnológico Ciencias de la Salud (PTS), Avenida de la Ilustracion 114, 18016 Granada, Spain.
Giorgia SantilliUniversity College London (UCL) Great Ormond Street Institute of Child Health (ICH), 30 Guilford Street, WC1N 1EH London, UK.
Anne GalyGenethon, 91000 Evry, France.
Adrian J ThrasherUniversity College London (UCL) Great Ormond Street Institute of Child Health (ICH), 30 Guilford Street, WC1N 1EH London, UK.
Francisco MartinGenomic Medicine Department, GENYO, Centre for Genomics and Oncological Research, Pfizer-University of Granada-Andalusian Regional Government, Parque Tecnológico Ciencias de la Salud (PTS), Avenida de la Ilustracion 114, 18016 Granada, Spain.
Pfizer-University of Granada-Junta de Andalucía Centre for Genomics and Oncological Research · ESUniversity College London · GBGenethon (France) · FR

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Transplant of gene-modified autologous hematopoietic progenitors cells has emerged as a new therapeutic approach for Wiskott-Aldrich syndrome (WAS), a primary immunodeficiency with microthrombocytopenia and abnormal lymphoid and myeloid functions. Despite the clinical benefits obtained in ongoing clinical trials, platelet restoration is suboptimal. The incomplete restoration of platelets in these patients can be explained either by a low number of corrected cells or by insufficient or inadequate WASP expression during megakaryocyte differentiation and/or in platelets. We therefore used

Indexed as

alternative WAS promotergene therapyhematopoietic stem and progenitor cellsHSPCslentiviral vectorsmegakaryocytic differentiationphenotypic rescuephysiollogical expressionWASKO cellular modelsWAS patientsWiskott-Aldrich syndrome

Identifiers

PMID33102615
PMCPMC7558809
OpenAlexW3087180166

What OpenQuestion holds

Textmetadata
LicenceCC BY
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.