Evidence map›Paper›PMID 30430274›Full record

ArticleMolecular and cellular pediatrics2018

Gene correction of HBB mutations in CD34

Justin S Antony, Ngadhnjim Latifi, A K M Ashiqul Haque, Andrés Lamsfus-Calle, Alberto Daniel-Moreno, Sebastian Graeter, Praveen Baskaran, Petra Weinmann, Markus Mezger, Rupert Handgretinger and 1 more

Open access · goldAbstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Molecular and cellular pediatrics, 2018. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 28 papers.

0numbers the graph read from it
0cells of the map it votes in
28citing papers in PubMed
2.8field-weighted citation impact, top 8% 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

28 citing papers in PubMed, 54 citations in OpenAlex.

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  6. Frontiers in cell and developmental biology · 2023
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  8. Prime editing in hematopoietic stem cells-FromFrontiers in genome editing · 2023
    Review
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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

11 authors at 2 institutions in 1 country.

Justin S AntonyDepartment of Pediatrics I, Pediatric Infectiology and Immunology, Translational Genomics and Gene Therapy in Pediatrics, University of Tuebingen, Tuebingen, Germany.
Ngadhnjim LatifiDepartment of Pediatrics I, Pediatric Infectiology and Immunology, Translational Genomics and Gene Therapy in Pediatrics, University of Tuebingen, Tuebingen, Germany.
A K M Ashiqul HaqueDepartment of Pediatrics I, Pediatric Infectiology and Immunology, Translational Genomics and Gene Therapy in Pediatrics, University of Tuebingen, Tuebingen, Germany.
Andrés Lamsfus-CalleUniversity Children's Hospital, Department of Pediatrics I, University of Tuebingen, Tuebingen, Germany.
Alberto Daniel-MorenoUniversity Children's Hospital, Department of Pediatrics I, University of Tuebingen, Tuebingen, Germany.
Sebastian GraeterDepartment of Pediatrics I, Pediatric Infectiology and Immunology, Translational Genomics and Gene Therapy in Pediatrics, University of Tuebingen, Tuebingen, Germany.
Praveen BaskaranCenter for Quantitative Biology, University of Tuebingen, Tuebingen, Germany.
Petra WeinmannDepartment of Pediatrics I, Pediatric Infectiology and Immunology, Translational Genomics and Gene Therapy in Pediatrics, University of Tuebingen, Tuebingen, Germany.
Markus MezgerUniversity Children's Hospital, Department of Pediatrics I, University of Tuebingen, Tuebingen, Germany.
Rupert HandgretingerUniversity Children's Hospital, Department of Pediatrics I, University of Tuebingen, Tuebingen, Germany.
Michael S D KormannDepartment of Pediatrics I, Pediatric Infectiology and Immunology, Translational Genomics and Gene Therapy in Pediatrics, University of Tuebingen, Tuebingen, Germany. michael.kormann@med.uni-tuebingen.de.ORCID http://orcid.org/0000-0003-3492-0393
University of Tübingen · DEUniversity Children's Hospital Tübingen · DE

Funding

fortüne grant 2412-0-0fortüne grant 2485-0-0H2020 European Research Council 637752HMZ Private Foundation NASanitätsrat Dr. Alexander Huebner und Gemahlin-Stiftung NA
6 · The paper itself

Abstract

backgroundβ-Thalassemia is an inherited hematological disorder caused by mutations in the human hemoglobin beta (HBB) gene that reduce or abrogate β-globin expression. Although lentiviral-mediated expression of β-globin and autologous transplantation is a promising therapeutic approach, the risk of insertional mutagenesis or low transgene expression is apparent. However, targeted gene correction of HBB mutations with programmable nucleases such as CRISPR/Cas9, TALENs, and ZFNs with non-viral repair templates ensures a higher safety profile and endogenous expression control.

methodsWe have compared three different gene-editing tools (CRISPR/Cas9, TALENs, and ZFNs) for their targeting efficiency of the HBB gene locus. As a proof of concept, we studied the personalized gene-correction therapy for a common β-thalassemia splicing variant HBB

resultsOur results exhibited that indel frequency of CRISPR/Cas9 was superior to TALENs and ZFNs (P < 0.0001). Our designed sgRNA targeting the site of HBB

conclusionOur approach provides guidance on non-viral gene correction in CD34

Indexed as

Beta-thalassemiaCas9 mRNACRISPR/Cas9Gene correctionHBBIVS1–110

Identifiers

PMID30430274
PMCPMC6236008
OpenAlexW2901108437

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.