ArticleScience advances2020
CRISPR-based gene editing enables
Open access · goldAbstract read
In one paragraph
Article in Science advances, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 74 papers, 1 of them a synthesis that pooled it.
0numbers the graph read from it
0cells of the map it votes in
74citing papers in PubMed, 1 pooled it
6.6field-weighted citation impact, top 2% of its field
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.
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.
Who cites it
74 citing papers in PubMed, 1 synthesis or guideline pooled it, 112 citations in OpenAlex.
- Persistence of CRISPR/Cas9 gene edited hematopoietic stem cells following transplantation: A systematic review and meta-analysis of preclinical studies.Stem cells translational medicine · 2021Pooled it
- Gene editing of hematopoietic stem cells: applications and advances.International journal of hematology · 2026Review
- Engraftment Outcome of CRISPR/Cas9-Edited Hematopoietic Stem Cells for Genetic Diseases: A Systematic Review and Meta-Analysis of Preclinical Evidence.Journal of hematology · 2026Article
- CRISPR-Cas9-based gene editing as a proof-of-concept approach in an inborn error of immunity caused by a DCLRE1C variant.Immunologic research · 2026Article
- Regulatory T Cells and Nanomaterials: Dual Perspectives in Therapeutics and Immunomodulation.Small science · 2026Review
- Regulatory T cells in the era of cell and gene therapy: biology, clinical translation, and future prospects.Frontiers in immunology · 2026Review
- Inborn errors of regulatory T-cell differentiation and function.The Journal of allergy and clinical immunology · 2025Review
- Precision T cell correction platform for inborn errors of immunity.Molecular therapy : the journal of the American Society of Gene Therapy · 2025Article
- Regulatory T Cell-Based Adoptive Cell Therapy in Autoimmunity.International journal of molecular sciences · 2025Review
- Gene therapy for inborn errors of immunity: past progress, current status and future directions.Rare disease and orphan drugs journal · 2025Article
- CRISPR tools for T cells: targeting the genome, epigenome, and transcriptome.Trends in cancer · 2025Review
- Advancements in CRISPR/Cas systems for disease treatment.Acta pharmaceutica Sinica. B · 2025Review
- Ex vivo modification of hematopoietic stem and progenitor cells for gene therapy.Molecular therapy : the journal of the American Society of Gene Therapy · 2025Review
- Mini-Review: Tregs as a Tool for Therapy-Obvious and Non-Obvious Challenges and Solutions.Cells · 2024Review
- Cellular and molecular basis of proximal small intestine disorders.Nature reviews. Gastroenterology & hepatology · 2024Review
- DNA-PK inhibition enhances gene editing efficiency in HSPCs for CRISPR-based treatment of X-linked hyper IgM syndrome.Molecular therapy. Methods & clinical development · 2024Article
- Rabies Virus Regulates Inflammatory Response in BV-2 Cells through Activation of Myd88 and NF-κB Signaling Pathways via TLR7.International journal of molecular sciences · 2024Article
- Induction of FoxP3 Pre-mRNA Alternative Splicing to Enhance the Suppressive Activity of Regulatory T Cells from Amyotrophic Lateral Sclerosis Patients.Biomedicines · 2024Article
- Genetically corrected RAG2-SCID human hematopoietic stem cells restore V(D)J-recombinase and rescue lymphoid deficiency.Blood advances · 2024Article
- Mending RAG2: gene editing for treatment of RAG2 deficiency.Blood advances · 2024Article
14 more citing papers are in PubMed but not listed here.
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
22 authors at 8 institutions in 5 countries.
M GoodwinDivision of Stem Cell Transplantation and Regenerative Medicine, Department of Pediatrics, Stanford University School of Medicine, Stanford, CA, USA.ORCID 0000-0002-6718-7610
E LeeDivision of Stem Cell Transplantation and Regenerative Medicine, Department of Pediatrics, Stanford University School of Medicine, Stanford, CA, USA.ORCID 0000-0002-0789-9149
U LakshmananDivision of Stem Cell Transplantation and Regenerative Medicine, Department of Pediatrics, Stanford University School of Medicine, Stanford, CA, USA.
S ShippDivision of Stem Cell Transplantation and Regenerative Medicine, Department of Pediatrics, Stanford University School of Medicine, Stanford, CA, USA.ORCID 0000-0003-3184-0435
L FroesslDivision of Stem Cell Transplantation and Regenerative Medicine, Department of Pediatrics, Stanford University School of Medicine, Stanford, CA, USA.ORCID 0000-0003-0569-4278
F BarzaghiDivision of Regenerative Medicine, Stem Cells and Gene Therapy, San Raffaele Telethon Institute for Gene Therapy, San Raffaele Scientific Institute, Milan, Italy.
L PasseriniDivision of Regenerative Medicine, Stem Cells and Gene Therapy, San Raffaele Telethon Institute for Gene Therapy, San Raffaele Scientific Institute, Milan, Italy.ORCID 0000-0002-4020-7386
M NarulaDivision of Stem Cell Transplantation and Regenerative Medicine, Department of Pediatrics, Stanford University School of Medicine, Stanford, CA, USA.
A SheikaliDivision of Stem Cell Transplantation and Regenerative Medicine, Department of Pediatrics, Stanford University School of Medicine, Stanford, CA, USA.
C M LeeAPC Microbiome Ireland, University College Cork, Cork, Ireland.
G BaoDepartment of Bioengineering, George R. Brown School of Engineering, Rice University, Houston, TX, USA.
C S BauerPhoenix Children's Hospital, Phoenix, AZ, USA.ORCID 0000-0002-4513-4577
H K MillerPhoenix Children's Hospital, Phoenix, AZ, USA.
M Garcia-LloretDivision of Immunology, Allergy, and Rheumatology, Department of Pediatrics, University of California, Los Angeles, Los Angeles, CA, USA.
M J ButteDivision of Immunology, Allergy, and Rheumatology, Department of Pediatrics, University of California, Los Angeles, Los Angeles, CA, USA.ORCID 0000-0002-4490-5595
A BertainaDivision of Stem Cell Transplantation and Regenerative Medicine, Department of Pediatrics, Stanford University School of Medicine, Stanford, CA, USA.ORCID 0000-0002-3729-436X
A ShahDivision of Stem Cell Transplantation and Regenerative Medicine, Department of Pediatrics, Stanford University School of Medicine, Stanford, CA, USA.ORCID 0000-0001-5700-6188
M Pavel-DinuDivision of Stem Cell Transplantation and Regenerative Medicine, Department of Pediatrics, Stanford University School of Medicine, Stanford, CA, USA.ORCID 0000-0003-2719-2498
A HendelDivision of Stem Cell Transplantation and Regenerative Medicine, Department of Pediatrics, Stanford University School of Medicine, Stanford, CA, USA.
M PorteusDivision of Stem Cell Transplantation and Regenerative Medicine, Department of Pediatrics, Stanford University School of Medicine, Stanford, CA, USA.ORCID 0000-0002-3850-4648
M G RoncaroloDivision of Stem Cell Transplantation and Regenerative Medicine, Department of Pediatrics, Stanford University School of Medicine, Stanford, CA, USA.
R BacchettaDivision of Stem Cell Transplantation and Regenerative Medicine, Department of Pediatrics, Stanford University School of Medicine, Stanford, CA, USA.ORCID 0000-0002-0606-6268
Stanford University · USCalifornia Institute for Regenerative Medicine · USPhoenix Children's Hospital · USThe San Raffaele Telethon Institute for Gene Therapy · ITUniversity of California, Los Angeles · USBar-Ilan University · ILRice University · USUniversity College Cork · IE
Funding
Stanford Center for Clinical & Translational Education and Research (Spectrum)UL1TR003142 · NCATS · STANFORD UNIVERSITY · PI O'HARA, RUTH M · 2019 to 2023
$45.0MSpectrum Stanford Center for clinical and Translational Research and EducationUL1TR001085 · NCATS · STANFORD UNIVERSITY · PI CULLEN, MARK RICHARD, GREENBERG, HARRY BERNARD · 2013 to 2017
$37.1MStoP STRokE: Sex-Specific Prevention of Stroke Through Reproductive Risk Factors and Sex Hormone EffectsK23HL140081 · NHLBI · RHODE ISLAND HOSPITAL · PI MADSEN, TRACY · 2018 to 2022
$945kGenome Editing to rescue FOXP3 Deficiency in IPEX syndromeR21AI123896 · NIAID · STANFORD UNIVERSITY · PI BACCHETTA, ROSA, PORTEUS, MATTHEW H · 2017 to 2018
$439kDirected Evolution of Peptide Inhibitors of Myc-Max Dimerization (PQ18)R21CA170721 · NCI · FRED HUTCHINSON CANCER RESEARCH CENTER · PI EISENMAN, ROBERT NEIL · 2012 to 2013
$410kNCATS NIH HHS UL1 TR001085NCATS NIH HHS UL1 TR003142NCI NIH HHS R21 CA170721NHLBI NIH HHS K23 HL140081NIAID NIH HHS R21 AI123896
Abstract
The prototypical genetic autoimmune disease is immune dysregulation polyendocrinopathy enteropathy X-linked (IPEX) syndrome, a severe pediatric disease with limited treatment options. IPEX syndrome is caused by mutations in the forkhead box protein 3 (
Indexed as
Gene EditingGenetic Diseases, X-LinkedChildForkhead Transcription FactorsHumansMutationPhenotypeT-Lymphocytes, RegulatoryForkhead Transcription FactorsFOXP3 protein, human
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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.