ReviewHuman gene therapy2012
Gene therapy for primary immunodeficiencies.
Review in Human gene therapy, 2012. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 30 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
30 citing papers in PubMed, 59 citations in OpenAlex.
- Trial
- The burden of severe leukocyte adhesion deficiency, type I (LAD-I): A multiple-case study from the caregiver's perspective.Journal of human immunity · 2026Article
- Precision diagnostics in children.Cambridge prisms. Precision medicine · 2023Review
- Review
- Molecular Evidence of Genome Editing in a Mouse Model of Immunodeficiency.Scientific reports · 2018Article
- Article
- Outcomes in two Japanese adenosine deaminase-deficiency patients treated by stem cell gene therapy with no cytoreductive conditioning.Journal of clinical immunology · 2015Article
- Large-scale clinical-grade retroviral vector production in a fixed-bed bioreactor.Journal of immunotherapy (Hagerstown, Md. : 1997) · 2015Article
- Clinical applications of gene therapy for primary immunodeficiencies.Human gene therapy · 2015Review
- The JAK-STAT pathway: impact on human disease and therapeutic intervention.Annual review of medicine · 2015Review
- Thymidine phosphorylase is both a therapeutic and a suicide gene in a murine model of mitochondrial neurogastrointestinal encephalomyopathy.Gene therapy · 2014Article
- Gene and cell therapy for children--new medicines, new challenges?Advanced drug delivery reviews · 2014Review
- The case for mandatory newborn screening for severe combined immunodeficiency (SCID).Journal of clinical immunology · 2014Review
- Bimodal high-affinity association of Brd4 with murine leukemia virus integrase and mononucleosomes.Nucleic acids research · 2014Article
- Efficiency and safety of O⁶-methylguanine DNA methyltransferase (MGMT(P140K))-mediated in vivo selection in a humanized mouse model.Human gene therapy · 2014Article
- Generation of X-CGD cells for vector evaluation from healthy donor CD34(+) HSCs by shRNA-mediated knock down of gp91(phox).Molecular therapy. Methods & clinical development · 2014Article
- Molecular mechanisms of retroviral integration site selection.Nucleic acids research · 2014Article
- Gene-modified hematopoietic stem cells for cancer immunotherapy.Human vaccines & immunotherapeutics · 2014Article
- Gene therapy on the move.EMBO molecular medicine · 2013Review
- Allogeneic blood and bone marrow cells for the treatment of severe epidermolysis bullosa: repair of the extracellular matrix.Lancet (London, England) · 2013Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
4 authors at 2 institutions in 2 countries.
Funding
Abstract
For over 40 years, primary immunodeficiencies (PIDs) have featured prominently in the development and refinement of human allogeneic hematopoietic stem cell transplantation. More recently, ex vivo somatic gene therapy using autologous cells has provided remarkable evidence of clinical efficacy in patients without HLA-matched stem cell donors and in whom toxicity of allogeneic procedures is likely to be high. Together with improved preclinical models, a wealth of information has accumulated that has allowed development of safer, more sophisticated technologies and protocols that are applicable to a much broader range of diseases. In this review we summarize the status of these gene therapy trials and discuss the emerging application of similar strategies to other PIDs.
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.