Evidence map›Paper›PMID 34801643›Full record

ReviewExperimental hematology2022

Translational research for bone marrow failure patients.

Camille Malouf, Stephen J Loughran, Adam C Wilkinson, Akiko Shimamura, Paula Río

Open access · bronzeAbstract readReview
In one paragraph

Review in Experimental hematology, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 3 papers.

0numbers the graph read from it
0cells of the map it votes in
3citing 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

3 citing papers in PubMed, 4 citations in OpenAlex.

  1. Article
  2. Review
  3. 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 5 institutions in 3 countries.

Camille MaloufCentre for Regenerative Medicine, University of Edinburgh, Edinburgh, UK.
Stephen J LoughranWellcome-MRC Cambridge Stem Cell Institute, University of Cambridge, Cambridge, UK.
Adam C WilkinsonMRC Weatherall Institute of Molecular Medicine, University of Oxford, Oxford, UK.
Akiko ShimamuraBone Marrow Failure and Myelodysplastic Syndrome Program, Dana-Farber/Boston Children's Cancer and Blood Disorders Center, Harvard Medical School, Boston, MA.
Paula RíoDivision of Hematopoietic Innovative Therapies, Centro de Investigaciones Energéticas Medioambientales y Tecnológicas (CIEMAT), Madrid, Spain; Centro de Investigación Biomédica en Red de Enfermedades Raras (CIBERER-ISCIII), Madrid, Spain; Advanced Therapies Unit, Instituto de Investigación Sanitaria Fundación Jiménez Díaz (IIS-FJD/UAM), Madrid, Spain.
Dana-Farber/Boston Children's Cancer and Blood Disorders Center · USInstituto de Salud Carlos III · ESMRC Centre for Regenerative Medicine · GBUniversity of Oxford · GBWellcome/MRC Cambridge Stem Cell Institute · GB

Funding

Systems Biology of Bone Marrow Failure and MDS for Precision MedicineRC2DK122533 · NIDDK · BOSTON CHILDREN'S HOSPITAL · PI FIGUEROA, MARIA EUGENIA, FLEMING, MARK D · 2019 to 2023
$6.4M
Medical Research Council MC_UU_00016/18NIDDK NIH HHS RC2 DK122533
6 · The paper itself

Abstract

Bone marrow failure syndromes encompass a range of inherited and acquired hematological diseases that result in insufficient blood cell production, which leads to severe complications including anemia, weakening of the immune system, impaired coagulation, and increased risk of cancer. Within inherited bone marrow failure syndromes, a number of genetically distinct diseases have been described including Shwachman-Diamond syndrome and Fanconi anemia. Given the genetic complexity and poor prognosis of these inherited bone marrow failure syndromes, there is increasing interest in both characterizing the genetic landscapes of these diseases and developing novel gene therapies to effectively monitor and cure patients. These topics were the focus of the winter 2021 International Society for Experimental Hematology New Investigator Webinar, which featured presentations by Dr. Akiko Shimamura and Dr. Paula Río. Here, we review the topics covered within this webinar.

Indexed as

AnimalsBone Marrow Failure DisordersClonal EvolutionFanconi AnemiaGenetic TherapyHumansShwachman-Diamond SyndromeTranslational Research, Biomedical

Identifiers

PMID34801643
PMCPMC9165438
OpenAlexW3213740893

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.