Evidence map›Paper›PMID 39460845›Full record

ArticleHuman cell2024

Assessment of non-myelotoxic agents as a preparatory regimen for hematopoietic stem cell gene therapy.

Mehmet Emin Şeker, Özgür Doğuş Erol, Burcu Pervin, Gerard Wagemaker, Niek P van Til, Fatima Aerts-Kaya

Abstract read
PubMed Publisher
In one paragraph

Article in Human cell, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 1 paper.

0numbers the graph read from it
0cells of the map it votes in
1citing papers in PubMed
–field-weighted citation impact
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

1 citing paper in PubMed.

  1. 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.

Mehmet Emin ŞekerDepartment of Stem Cell Sciences, Graduate School of Health Sciences, Hacettepe University, Ankara, Turkey.ORCID http://orcid.org/0000-0002-8240-5938
Özgür Doğuş ErolDepartment of Stem Cell Sciences, Graduate School of Health Sciences, Hacettepe University, Ankara, Turkey.ORCID http://orcid.org/0000-0001-9301-5401
Burcu PervinDepartment of Stem Cell Sciences, Graduate School of Health Sciences, Hacettepe University, Ankara, Turkey.ORCID http://orcid.org/0000-0002-5866-0955
Gerard WagemakerCenter for Stem Cell Research and Development, Hacettepe University, Ankara, Turkey.ORCID http://orcid.org/0000-0001-7898-5178
Niek P van TilAmsterdam Leukodystrophy Center, Emma Children's Hospital, Amsterdam University Medical Center, Amsterdam Neuroscience, 1081 HV, Amsterdam, The Netherlands.ORCID http://orcid.org/0000-0002-9515-4103
Fatima Aerts-KayaDepartment of Stem Cell Sciences, Graduate School of Health Sciences, Hacettepe University, Ankara, Turkey. fatima.aerts@hacettepe.edu.tr.ORCID http://orcid.org/0000-0002-9583-8572

Funding

European Union Framework Program 7 HEALTH-2010-261387Hacettepe Üniversitesi TAY2022-20436Hacettepe Üniversitesi TUK-2019-17760Scientific and Technological Research Council of Turkey (TÜBİTAK) 1002-220S338Scientific and Technological Research Council of Turkey (TÜBİTAK) 2221-2017/2
6 · The paper itself

Abstract

RAG2 deficiency is characterized by a lack of B and T lymphocytes, causing severe lethal infections. Currently, RAG2 deficiency is treated with a Hematopoietic Stem Cell transplantation (HSCT). Most conditioning regimens used before HSCT consist of alkylating myelotoxic agents with or without irradiation and affect growth and development of pediatric patients. Here, we developed a non-myelotoxic regimen using G-CSF, VLA-4I or AMD3100. These agents are known HSC mobilizers or affect bone marrow (BM) permeability and may support the homing of HSCs to the BM, without inducing major side effects. Female Rag2

Indexed as

BenzylaminesCyclamsGenetic TherapyGranulocyte Colony-Stimulating FactorHematopoietic Stem Cell TransplantationAnimalsBusulfanDNA-Binding ProteinsFemaleHematopoietic Stem CellsHeterocyclic CompoundsHumansMaleMiceMice, Inbred C57BLTransplantation ConditioningBenzylaminesBusulfanCyclamsDNA-Binding ProteinsGranulocyte Colony-Stimulating FactorHeterocyclic CompoundsplerixaforRag2 protein, mouseAMD3100ConditioningG-CSFGene therapyHematopoietic stem cell transplantationRAG2VLA-4I

Identifiers

PMID39460845

What OpenQuestion holds

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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.