ArticleFrontiers in immunology2022
CRISPR-Cas9-AAV versus lentivector transduction for genome modification of X-linked severe combined immunodeficiency hematopoietic stem cells.
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In one paragraph
Article in Frontiers in immunology, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 20 papers, 1 of them a synthesis that pooled it.
0numbers the graph read from it
0cells of the map it votes in
20citing papers in PubMed, 1 pooled it
7.5field-weighted citation impact, top 2% of its field
What it found
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Who cites it
20 citing papers in PubMed, 1 synthesis or guideline pooled it, 24 citations in OpenAlex.
- Newborn screening for severe combined immunodeficiency in the NHS newborn blood spot screening programme: a systematic review.Health technology assessment (Winchester, England) · 2026Pooled it
- Delivery Systems for Therapeutic Genome Editing: Challenges, Innovations, and Future Perspectives.MedComm · 2026Review
- CRISPR Gene Tagging for Illuminating Endogenous Protein Dynamics.International journal of molecular sciences · 2026Review
- Modeling and correction of SCID-X1 using CRISPR-Cas9 homology-directed repair in human HSPCs.Molecular therapy. Nucleic acids · 2026Article
- 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
- Review
- IL2RG-related immunodeficiencies: from SCID to atypical presentations.Frontiers in immunology · 2026Review
- CRISPR-based therapeutic genome editing for inherited blood disorders.Nature reviews. Drug discovery · 2025Review
- Integrase-Deficient Lentiviral Vector as a Platform for Efficient CRISPR/Cas9-Mediated Gene Editing for Mucopolysaccharidosis IVA.International journal of molecular sciences · 2025Article
- Ligand-modified rAAV6 vectors with nanoblades allow high-level gene knockin in HSPCs without compromising cell survival.Molecular therapy. Nucleic acids · 2025Article
- Donor insertion into CX3CR1 allows epigenetic modulation of a constitutive promoter on hematopoietic stem cells and its activation upon myeloid differentiation.Nucleic acids research · 2025Article
- High-fidelity PAMless base editing of hematopoietic stem cells to treat chronic granulomatous disease.Science translational medicine · 2024Article
- Rediscovering the human thymus through cutting-edge technologies.The Journal of experimental medicine · 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
- Mending RAG2: gene editing for treatment of RAG2 deficiency.Blood advances · 2024Article
- Impact of CRISPR/HDR editing versus lentiviral transduction on long-term engraftment and clonal dynamics of HSPCs in rhesus macaques.Cell stem cell · 2024Article
- Transient inhibition of 53BP1 increases the frequency of targeted integration in human hematopoietic stem and progenitor cells.Nature communications · 2024Article
- NovelCentral-European journal of immunology · 2024Review
- Advances in CRISPR-Cas technology and its applications: revolutionising precision medicine.Frontiers in genome editing · 2024Review
- Homology-Directed-Repair-Based Genome Editing in HSPCs for the Treatment of Inborn Errors of Immunity and Blood Disorders.Pharmaceutics · 2023Review
Corrections and comments
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Authors and funding
21 authors at 9 institutions in 1 country.
Julie BraultLaboratory of Clinical Immunology and Microbiology, National Institute of Allergy and Infectious Diseases (NIAID), National Institutes of Health (NIH), Bethesda, MD, United States.
Taylor LiuLaboratory of Clinical Immunology and Microbiology, National Institute of Allergy and Infectious Diseases (NIAID), National Institutes of Health (NIH), Bethesda, MD, United States.
Siyuan LiuCancer Research Technology Program, Leidos Biomedical Research Inc., Frederick, MD, United States.
Amanda LawsonLaboratory of Clinical Immunology and Microbiology, National Institute of Allergy and Infectious Diseases (NIAID), National Institutes of Health (NIH), Bethesda, MD, United States.
Uimook ChoiLaboratory of Clinical Immunology and Microbiology, National Institute of Allergy and Infectious Diseases (NIAID), National Institutes of Health (NIH), Bethesda, MD, United States.
Nikita KozhushkoLaboratory of Clinical Immunology and Microbiology, National Institute of Allergy and Infectious Diseases (NIAID), National Institutes of Health (NIH), Bethesda, MD, United States.
Vera BzhilyanskayaLaboratory of Clinical Immunology and Microbiology, National Institute of Allergy and Infectious Diseases (NIAID), National Institutes of Health (NIH), Bethesda, MD, United States.
Mara Pavel-DinuDepartment of Pediatrics, Division of Stem Cell Transplantation and Regenerative Medicine, Stanford University, Palo Alto, CA, United States.
Ronald J MeisCellscript LLC Inc., Madison, WI, United States.
Michael A EckhausDivision of Veterinary Resources, Office of Research Services, National Institutes of Health, Bethesda, MD, United States.
Sandra S BurkettMolecular Cytogenetic Core Facility, Center for Cancer Research, National Cancer Institute, National Institutes of Health, Frederick, MD, United States.
Marita BosticardoLaboratory of Clinical Immunology and Microbiology, National Institute of Allergy and Infectious Diseases (NIAID), National Institutes of Health (NIH), Bethesda, MD, United States.
Benjamin P KleinstiverCenter for Genomic Medicine and Department of Pathology, Massachusetts General Hospital, Boston, MA, United States.
Luigi D NotarangeloLaboratory of Clinical Immunology and Microbiology, National Institute of Allergy and Infectious Diseases (NIAID), National Institutes of Health (NIH), Bethesda, MD, United States.
Cicera R LazzarottoDepartment of Hematology, St. Jude Children's Research Hospital, Memphis, TN, United States.
Shengdar Q TsaiDepartment of Hematology, St. Jude Children's Research Hospital, Memphis, TN, United States.
Xiaolin WuCancer Research Technology Program, Leidos Biomedical Research Inc., Frederick, MD, United States.
Gary A DahlCellscript LLC Inc., Madison, WI, United States.
Matthew H PorteusDepartment of Pediatrics, Division of Stem Cell Transplantation and Regenerative Medicine, Stanford University, Palo Alto, CA, United States.
Harry L MalechLaboratory of Clinical Immunology and Microbiology, National Institute of Allergy and Infectious Diseases (NIAID), National Institutes of Health (NIH), Bethesda, MD, United States.
Suk See De RavinLaboratory of Clinical Immunology and Microbiology, National Institute of Allergy and Infectious Diseases (NIAID), National Institutes of Health (NIH), Bethesda, MD, United States.
National Institute of Allergy and Infectious Diseases · USNational Institutes of Health · USStanford University · USSt. Jude Children's Research Hospital · USHarvard University · USLeidos Biomedical Research Inc. (United States)Leidos (United States) · USNational Cancer Institute · USOffice of Research Services · US
Funding
PROJECT 4: Somatic evolution of the hematopoietic system in cardiovascular diseaseP01HL142494 · NHLBI · MASSACHUSETTS GENERAL HOSPITAL · PI Matthias Nahrendorf · 2019 to 2026
$19.5MPERIPHERAL BLOOD PROGENITORS-TARGET FOR GENE TRANSFERZ01AI000644 · NIAID · NIAID EXTRAMURAL ACTIVITIES · PI MALECH, HARRY L · 1992 to 2008
$4.3MA novel human T-cell platform to define biological effects of genome editingU01AI157189 · NIAID · ST. JUDE CHILDREN'S RESEARCH HOSPITAL · PI TSAI, SHENGDAR · 2020 to 2022
$1.8MOptimizing Cancer Immunotherapy Safety and Efficacy using Genome EditingR00CA218870 · NCI · MASSACHUSETTS GENERAL HOSPITAL · PI KLEINSTIVER, BENJAMIN PETER · 2018 to 2020
$730kBasic Research on Hematopoietic Adult Human Stem CellsZ01AI000988 · NIAID · NIAID EXTRAMURAL ACTIVITIES · PI MALECH, HARRY L · 2007 to 2008
$669kIntramural NIH HHS Z01 AI000644Intramural NIH HHS Z01 AI000988NCI NIH HHS R00 CA218870NHLBI NIH HHS P01 HL142494NIAID NIH HHS U01 AI157189
Abstract
Introduction: Methods: We compare the two approaches for integration of Results and discussion:
Indexed as
X-Linked Combined Immunodeficiency DiseasesAnimalsCRISPR-Cas SystemsDependovirusHematopoietic Stem CellsMiceMice, SCIDAAV6CRISPR-Cas9lentivectorpreclinical studiesXSCID
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LicenceCC BY
Read underepoch 390
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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.