ReviewFrontiers in immunology2015
Novel Genome-Editing Tools to Model and Correct Primary Immunodeficiencies.
Review in Frontiers in immunology, 2015. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 15 papers.
What it found
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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
15 citing papers in PubMed, 38 citations in OpenAlex.
- Modeling myeloid cell development in health and disease using induced pluripotent stem cells.Frontiers in immunology · 2026Review
- Review
- A variant in human AIOLOS impairs adaptive immunity by interfering with IKAROS.Nature immunology · 2021Article
- Review
- Applications of genome editing technology in the targeted therapy of human diseases: mechanisms, advances and prospects.Signal transduction and targeted therapy · 2020Review
- Genome editing: A perspective on the application of CRISPR/Cas9 to study human diseases (Review).International journal of molecular medicine · 2019Review
- Programmable Molecular Scissors: Applications of a New Tool for Genome Editing in Biotech.Molecular therapy. Nucleic acids · 2019Review
- Applications of Genome Editing Technology in Animal Disease Modeling and Gene Therapy.Computational and structural biotechnology journal · 2019Review
- Rare Genetic Blood Disease Modeling in Zebrafish.Frontiers in genetics · 2018Review
- Targeted genome editing restores T cell differentiation in a humanized X-SCID pluripotent stem cell disease model.Scientific reports · 2017Article
- Genetic Engineering and Manufacturing of Hematopoietic Stem Cells.Molecular therapy. Methods & clinical development · 2017Review
- To CRISPR and beyond: the evolution of genome editing in stem cells.Regenerative medicine · 2016Review
- mRNA trans-splicing in gene therapy for genetic diseases.Wiley interdisciplinary reviews. RNA · 2016Review
- Hematopoietic Stem Cells in Regenerative Medicine: Astray or on the Path?Transfusion medicine and hemotherapy : offizielles Organ der Deutschen Gesellschaft fur Transfusionsmedizin und Immunhamatologie · 2016Review
- Flow Cytometry, a Versatile Tool for Diagnosis and Monitoring of Primary Immunodeficiencies.Clinical and vaccine immunology : CVI · 2016Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
3 authors at 3 institutions in 4 countries.
Funding
Abstract
Severe combined immunodeficiency (SCID) and other severe non-SCID primary immunodeficiencies (non-SCID PID) can be treated by allogeneic hematopoietic stem cell (HSC) transplantation, but when histocompatibility leukocyte antigen-matched donors are lacking, this can be a high-risk procedure. Correcting the patient's own HSCs with gene therapy offers an attractive alternative. Gene therapies currently being used in clinical settings insert a functional copy of the entire gene by means of a viral vector. With this treatment, severe complications may result due to integration within oncogenes. A promising alternative is the use of endonucleases such as ZFNs, TALENs, and CRISPR/Cas9 to introduce a double-stranded break in the DNA and thus induce homology-directed repair. With these genome-editing tools a correct copy can be inserted in a precisely targeted "safe harbor." They can also be used to correct pathogenic mutations in situ and to develop cellular or animal models needed to study the pathogenic effects of specific genetic defects found in immunodeficient patients. This review discusses the advantages and disadvantages of these endonucleases in gene correction and modeling with an emphasis on CRISPR/Cas9, which offers the most promise due to its efficacy and versatility.
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Registered trials
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.