ReviewCurrent opinion in hematology2008
Recent advances in gene therapy for severe congenital immunodeficiency diseases.
Review in Current opinion in hematology, 2008. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 12 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
12 citing papers in PubMed, 40 citations in OpenAlex.
- Ciliopathies in pediatric endocrinology.Annals of pediatric endocrinology & metabolism · 2023Article
- Current and Future Treatments in Primary Ciliary Dyskinesia.International journal of molecular sciences · 2021Review
- Proceedings of the 2nd BEAT-PCD conference and 3rd PCD training school: part 1.BMC proceedings · 2018Article
- Physiological regulation of transgene expression by a lentiviral vector containing the A2UCOE linked to a myeloid promoter.Gene therapy · 2012Article
- The function of dog models in developing gene therapy strategies for human health.Mammalian genome : official journal of the International Mammalian Genome Society · 2011Review
- Gene therapy of chronic granulomatous disease: the engraftment dilemma.Molecular therapy : the journal of the American Society of Gene Therapy · 2011Review
- Scavenger receptors and their potential as therapeutic targets in the treatment of cardiovascular disease.International journal of hypertension · 2010Article
- The clinical utility of molecular diagnostic testing for primary immune deficiency disorders: a case based review.Allergy, asthma, and clinical immunology : official journal of the Canadian Society of Allergy and Clinical Immunology · 2010Article
- Progress and prospects: gene therapy for inherited immunodeficiencies.Gene therapy · 2009Review
- Evidence for long-term efficacy and safety of gene therapy for Wiskott-Aldrich syndrome in preclinical models.Molecular therapy : the journal of the American Society of Gene Therapy · 2009Article
- Inborn errors of purine and pyrimidine metabolism.Journal of inherited metabolic disease · 2009Review
- Partially corrected X-linked severe combined immunodeficiency: long-term problems and treatment options.Immunologic research · 2009Review
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 2 institutions in 1 country.
Funding
Abstract
purpose of reviewTo discuss new data on the safety and efficacy of the ongoing gene therapy trials for primary immune deficiencies, the first reports of new trials and the preclinical developments that are likely to be translated to the clinic in the near future. RECENT
findingsBoth clinical successes and severe adverse events continue to be reported in trials of gammaretroviral gene therapy for severe combined immune deficiency-X1, adenosine deaminase-deficient forms of severe combined immune deficiency and chronic granulomatous disease. Insertion site analyses of recently reported trials on all of these diseases have discovered preferential insertion in the 5' ends of genes, including potentially dangerous ones such as proto-oncogenes and signal transduction and proliferation genes. Preclinical work on rodent and canine models has tested novel vectors, including lentiviruses and foamy viruses. SUMMARY: Gene therapy for the most common forms of severe combined immune deficiency can lead to immune reconstitution in most patients, although a minority of patients has derived minimal clinical benefit and some have suffered severe adverse events including death. Ongoing preclinical work attempts to address the latter shortcoming. Meanwhile, in the presence of a careful risk-benefit assessment, gene therapy remains an appropriate subject of clinical investigation.
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.