ReviewThe Journal of allergy and clinical immunology2017
New frontiers in the therapy of primary immunodeficiency: From gene addition to gene editing.
Review in The Journal of allergy and clinical immunology, 2017. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 22 papers.
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
22 citing papers in PubMed, 46 citations in OpenAlex.
- Advancements in CRISPR/Cas systems for disease treatment.Acta pharmaceutica Sinica. B · 2025Review
- Article
- Antiviral options and therapeutics against influenza: history, latest developments and future prospects.Frontiers in cellular and infection microbiology · 2023Review
- Advances in CRISPR/Cas gene therapy for inborn errors of immunity.Frontiers in immunology · 2023Review
- CRISPR/Cas systems usher in a new era of disease treatment and diagnosis.Molecular biomedicine · 2022Review
- Using a scenario approach to assess for the current and future demand of immunoglobulins: An interview and literature study from The Netherlands.Transfusion medicine (Oxford, England) · 2022Review
- Gene Edited T Cell Therapies for Inborn Errors of Immunity.Frontiers in genome editing · 2022Review
- CRISPR-targeted MAGT1 insertion restores XMEN patient hematopoietic stem cells and lymphocytes.Blood · 2021Article
- Mutation-Specific Guide RNA for Compound Heterozygous Porphyria On-target Scarless Correction by CRISPR/Cas9 in Stem Cells.Stem cell reports · 2020Article
- Successful Preclinical Development of Gene Therapy for Recombinase-Activating Gene-1-Deficient SCID.Molecular therapy. Methods & clinical development · 2020Article
- Genome-edited adult stem cells: Next-generation advanced therapy medicinal products.Stem cells translational medicine · 2020Review
- Gene analysis of seven cases of primary immunodeficiency.Translational pediatrics · 2020Article
- Genome editing: A perspective on the application of CRISPR/Cas9 to study human diseases (Review).International journal of molecular medicine · 2019Review
- CRISPR-Cas9 genome editing induces megabase-scale chromosomal truncations.Nature communications · 2019Article
- Consensus approach for the management of severe combined immune deficiency caused by adenosine deaminase deficiency.The Journal of allergy and clinical immunology · 2019Review
- Newborn screening for severe combined immunodeficiency and T-cell lymphopenia.Immunological reviews · 2019Review
- Efficacy of lentivirus-mediated gene therapy in an Omenn syndrome recombination-activating gene 2 mouse model is not hindered by inflammation and immune dysregulation.The Journal of allergy and clinical immunology · 2018Article
- [Clinical features and gene mutations of primary immunodeficiency disease: an analysis of 7 cases].Zhongguo dang dai er ke za zhi = Chinese journal of contemporary pediatrics · 2018Article
- Manufacture of Third-Generation Lentivirus for Preclinical Use, with Process Development Considerations for Translation to Good Manufacturing Practice.Human gene therapy methods · 2018Article
- IL-6 receptor blockade corrects defects of XIAP-deficient regulatory T cells.Nature communications · 2018Article
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
2 authors at 2 institutions in 1 country.
Funding
Abstract
The most severe primary immune deficiency diseases (PIDs) have been successfully treated with allogeneic hematopoietic stem cell transplantation for more than 4 decades. However, such transplantations have the best outcomes when there is a well-matched donor available because immune complications, such as graft-versus-host disease, are greater without a matched sibling donor. Gene therapy has been developed as a method to perform autologous transplantations of a patient's own stem cells that are genetically corrected. Through an iterative bench-to-bedside-and-back process, methods to efficiently add new copies of the relevant gene to hematopoietic stem cells have led to safe and effective treatments for several PIDs, including forms of severe combined immune deficiency, Wiskott-Aldrich syndrome, and chronic granulomatous disease. New methods for gene editing might allow additional PIDs to be treated by gene therapy because they will allow the endogenous gene to be repaired and expressed under its native regulatory elements, which are essential for genes involved in cell processes of signaling, activation, and proliferation. Gene therapy is providing exciting new treatment options for patients with PIDs, and advances are sure to continue.
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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.