Evidence map›Paper›PMID 26321643›Full record

ArticleCell reports2015

Modeling Human Severe Combined Immunodeficiency and Correction by CRISPR/Cas9-Enhanced Gene Targeting.

Chia-Wei Chang, Yi-Shin Lai, Erik Westin, Alireza Khodadadi-Jamayran, Kevin M Pawlik, Lawrence S Lamb, Frederick D Goldman, Tim M Townes

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

Article in Cell reports, 2015. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 61 papers.

0numbers the graph read from it
0cells of the map it votes in
61citing papers in PubMed
6.8field-weighted citation impact, top 2% 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

61 citing papers in PubMed, 109 citations in OpenAlex.

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  19. Frontiers in immunology · 2022
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1 more citing papers are in PubMed but not listed here.

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

8 authors at 1 institution in 1 country.

Chia-Wei ChangDepartment of Biochemistry and Molecular Genetics, Schools of Medicine and Dentistry, University of Alabama at Birmingham, Birmingham, AL 35294, USA; UAB Stem Cell Institute, Schools of Medicine and Dentistry, University of Alabama at Birmingham, Birmingham, AL 35294, USA.
Yi-Shin LaiDepartment of Biochemistry and Molecular Genetics, Schools of Medicine and Dentistry, University of Alabama at Birmingham, Birmingham, AL 35294, USA; UAB Stem Cell Institute, Schools of Medicine and Dentistry, University of Alabama at Birmingham, Birmingham, AL 35294, USA.
Erik WestinDepartment of Biochemistry and Molecular Genetics, Schools of Medicine and Dentistry, University of Alabama at Birmingham, Birmingham, AL 35294, USA; UAB Stem Cell Institute, Schools of Medicine and Dentistry, University of Alabama at Birmingham, Birmingham, AL 35294, USA.
Alireza Khodadadi-JamayranDepartment of Biochemistry and Molecular Genetics, Schools of Medicine and Dentistry, University of Alabama at Birmingham, Birmingham, AL 35294, USA; UAB Stem Cell Institute, Schools of Medicine and Dentistry, University of Alabama at Birmingham, Birmingham, AL 35294, USA.
Kevin M PawlikDepartment of Biochemistry and Molecular Genetics, Schools of Medicine and Dentistry, University of Alabama at Birmingham, Birmingham, AL 35294, USA; UAB Stem Cell Institute, Schools of Medicine and Dentistry, University of Alabama at Birmingham, Birmingham, AL 35294, USA.
Lawrence S LambDepartment of Medicine, Division of Hematology/Oncology, Schools of Medicine and Dentistry, University of Alabama at Birmingham, Birmingham, AL 35294, USA; Cell Therapy Lab, Schools of Medicine and Dentistry, University of Alabama at Birmingham, Birmingham, AL 35294, USA; UAB Stem Cell Institute, Schools of Medicine and Dentistry, University of Alabama at Birmingham, Birmingham, AL 35294, USA.
Frederick D GoldmanDepartment of Pediatrics, Division of Hematology/Oncology, Schools of Medicine and Dentistry, University of Alabama at Birmingham, Birmingham, AL 35294, USA; UAB Stem Cell Institute, Schools of Medicine and Dentistry, University of Alabama at Birmingham, Birmingham, AL 35294, USA.
Tim M TownesDepartment of Biochemistry and Molecular Genetics, Schools of Medicine and Dentistry, University of Alabama at Birmingham, Birmingham, AL 35294, USA; UAB Stem Cell Institute, Schools of Medicine and Dentistry, University of Alabama at Birmingham, Birmingham, AL 35294, USA. Electronic address: ttownes@uab.edu.
University of Alabama at Birmingham · US

Funding

Genetic Modifers of Sickle Cell DiseaseR01HL068965 · NHLBI · UNIVERSITY OF ALABAMA AT BIRMINGHAM · PI TOWNES, TIM M. · 2001 to 2005
$2.9M
Gene Replacement Therapy in Induced Pluripotent Stem (iPS) Cells for Treatment ofR03HL096268 · NHLBI · UNIVERSITY OF ALABAMA AT BIRMINGHAM · PI TOWNES, TIM M. · 2009 to 2009
$36k
NHLBI NIH HHS R01 HL068965NHLBI NIH HHS R03 HL096268
6 · The paper itself

Abstract

Mutations of the Janus family kinase JAK3 gene cause severe combined immunodeficiency (SCID). JAK3 deficiency in humans is characterized by the absence of circulating T cells and natural killer (NK) cells with normal numbers of poorly functioning B cells (T(-)B(+)NK(-)). Using SCID patient-specific induced pluripotent stem cells (iPSCs) and a T cell in vitro differentiation system, we demonstrate a complete block in early T cell development of JAK3-deficient cells. Correction of the JAK3 mutation by CRISPR/Cas9-enhanced gene targeting restores normal T cell development, including the production of mature T cell populations with a broad T cell receptor (TCR) repertoire. Whole-genome sequencing of corrected cells demonstrates no CRISPR/Cas9 off-target modifications. These studies describe an approach for the study of human lymphopoiesis and provide a foundation for gene correction therapy in humans with immunodeficiencies.

Indexed as

Genetic TherapyBacterial ProteinsBase SequenceCells, CulturedChild, PreschoolClustered Regularly Interspaced Short Palindromic RepeatsCRISPR-Associated Protein 9DNA Mutational AnalysisEndonucleasesGene TargetingHumansInduced Pluripotent Stem CellsJanus Kinase 3MaleMutation, MissenseProto-Oncogene Proteins c-bcl-2Bacterial ProteinsBCL2 protein, humanCas9 protein, Francisella novicidaCRISPR-Associated Protein 9EndonucleasesJAK3 protein, humanJanus Kinase 3Proto-Oncogene Proteins c-bcl-2

Identifiers

PMID26321643
PMCPMC13101568
OpenAlexW1710674277

What OpenQuestion holds

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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.