ReviewCurrent allergy and asthma reports2016
Gene Therapy for the Treatment of Primary Immune Deficiencies.
Review in Current allergy and asthma reports, 2016. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 26 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
26 citing papers in PubMed, 62 citations in OpenAlex.
- Nanomedicine Solutions for Alzheimer's Disease: A Critical Review of Therapeutic Nanoparticle Strategies.ACS omega · 2025Review
- Review
- Targeted Next-Generation Sequencing in the Molecular Diagnosis of Severe Combined Immunodeficiency.Medicina (Kaunas, Lithuania) · 2025Article
- Inborn errors of immunity (primary immunodeficiencies).Allergy, asthma, and clinical immunology : official journal of the Canadian Society of Allergy and Clinical Immunology · 2025Review
- Gene Edited T Cell Therapies for Inborn Errors of Immunity.Frontiers in genome editing · 2022Review
- Improved functionality and potency of next generation BinMLV viral vectors toward safer gene therapy.Molecular therapy. Methods & clinical development · 2021Article
- Exploring genetic defects in adults who were clinically diagnosed as severe combined immune deficiency during infancy.Immunologic research · 2021Article
- Gene delivery using AAV8Molecular therapy. Methods & clinical development · 2021Article
- Optimizing lentiviral vector transduction of hematopoietic stem cells for gene therapy.Gene therapy · 2020Article
- Modulating gene regulation to treat genetic disorders.Nature reviews. Drug discovery · 2020Review
- Production and Application of Multicistronic Constructs for Various Human Disease Therapies.Pharmaceutics · 2019Review
- Review
- Fine-Tuning Cytokine Signals.Annual review of immunology · 2019Review
- Gene Therapy Leaves a Vicious Cycle.Frontiers in oncology · 2019Review
- Primary immunodeficiency.Allergy, asthma, and clinical immunology : official journal of the Canadian Society of Allergy and Clinical Immunology · 2018Review
- Anti-IgE therapy for IgE-mediated allergic diseases: from neutralizing IgE antibodies to eliminating IgEClinical and translational allergy · 2018Review
- Long-Term Outcome of Adenosine Deaminase-Deficient Patients-a Single-Center Experience.Journal of clinical immunology · 2017Article
- Genetic Engineering and Manufacturing of Hematopoietic Stem Cells.Molecular therapy. Methods & clinical development · 2017Review
- INSPIIRED: A Pipeline for Quantitative Analysis of Sites of New DNA Integration in Cellular Genomes.Molecular therapy. Methods & clinical development · 2017Article
- Treatment of infants identified as having severe combined immunodeficiency by means of newborn screening.The Journal of allergy and clinical immunology · 2017Article
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 1 institution in 1 country.
Funding
Abstract
The use of gene therapy in the treatment of primary immune deficiencies (PID) has advanced significantly in the last decade. Clinical trials for X-linked severe combined immunodeficiency, adenosine deaminase deficiency (ADA), chronic granulomatous disease, and Wiskott-Aldrich syndrome have demonstrated that gene transfer into hematopoietic stem cells and autologous transplant can result in clinical improvement and is curative for many patients. Unfortunately, early clinical trials were complicated by vector-related insertional mutagenic events for several diseases with the exception of ADA-deficiency SCID. These results prompted the current wave of clinical trials for primary immunodeficiency using alternative retro- or lenti-viral vector constructs that are self-inactivating, and they have shown clinical efficacy without leukemic events thus far. The field of gene therapy continues to progress, with improvements in viral vector profiles, stem cell culturing techniques, and site-specific genome editing platforms. The future of gene therapy is promising, and we are quickly moving towards a time when it will be a standard cellular therapy for many forms of PID.
Indexed as
Identifiers
What OpenQuestion holds
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.