ReviewThe Journal of clinical investigation2007
Gene therapy for severe combined immunodeficiency: are we there yet?
Review in The Journal of clinical investigation, 2007. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT03538899 (Phase I/II Safety and Efficacy Study of Gene Transfer for Artemis-Deficient Severe Combined Immunodeficiency), which is not on this map. Cited by 64 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.
Phase I/II Safety and Efficacy Study of Gene Transfer for Artemis-Deficient Severe Combined Immunodeficiency (ART-SCID) in Newly Diagnosed Patients Using Self-Inactivating Lentiviral Vector (AProArt) to Transduce Autologous CD34 Hematopoietic Cells
Who cites it
64 citing papers in PubMed, 225 citations in OpenAlex.
- Multilineage hematopoietic reconstitution without clonal selection in ADA-SCID patients treated with stem cell gene therapy.The Journal of clinical investigation · 2007Trial
- Translation of unspliced retroviral genomic RNA in the host cell is regulated in both space and time.The Journal of cell biology · 2025Article
- Differentiation ability of hematopoietic stem cells and mesenchymal stem cells isolated from human peripheral blood.Frontiers in cell and developmental biology · 2024Article
- A Multifaceted Approach to Optimizing AAV Delivery to the Brain for the Treatment of Neurodegenerative Diseases.Frontiers in neuroscience · 2021Article
- Successful Preclinical Development of Gene Therapy for Recombinase-Activating Gene-1-Deficient SCID.Molecular therapy. Methods & clinical development · 2020Article
- Darwinian selection within an individual or somatic selection: facts and models.Journal of molecular cell biology · 2019Article
- ADA Deficiency: Evaluation of the Clinical and Laboratory Features and the Outcome.Journal of clinical immunology · 2018Article
- Advances in gene therapy for muscular dystrophies.F1000Research · 2016Review
- Gene Therapy for Metabolic Diseases.Translational science of rare diseases · 2016Article
- Assessing risk/benefit for trials using preclinical evidence: a proposal.Journal of medical ethics · 2016Article
- Towards in vivo amplification: Overcoming hurdles in the use of hematopoietic stem cells in transplantation and gene therapy.World journal of stem cells · 2015Review
- [Heterogeneity of hematopoietic stem cell].Zhonghua xue ye xue za zhi = Zhonghua xueyexue zazhi · 2015Article
- Gene therapy for ocular diseases meditated by ultrasound and microbubbles (Review).Molecular medicine reports · 2015Review
- Article
- Review
- No evidence of clonal dominance after transplant of HOXB4-expanded cord blood cells in a nonhuman primate model.Experimental hematology · 2014Article
- MLV requires Tap/NXF1-dependent pathway to export its unspliced RNA to the cytoplasm and to express both spliced and unspliced RNAs.Retrovirology · 2014Article
- The era of genomic medicine.Clinical medicine (London, England) · 2013Review
- Therapy for Duchenne muscular dystrophy: renewed optimism from genetic approaches.Nature reviews. Genetics · 2013Review
- Gene therapy for cardiovascular disease mediated by ultrasound and microbubbles.Cardiovascular ultrasound · 2013Review
4 more citing papers are in PubMed but not listed here.
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
Inherited and acquired diseases of the hematopoietic system can be cured by allogeneic hematopoietic stem cell transplantation. This treatment strategy is highly successful when an HLA-matched sibling donor is available, but if not, few therapeutic options exist. Gene-modified, autologous bone marrow transplantation can circumvent the severe immunological complications that occur when a related HLA-mismatched donor is used and thus represents an attractive alternative. In this review, we summarize the advantages and limitations associated with the use of gene therapy to cure SCID. Insertional mutagenesis and technological improvements aimed at increasing the safety of this strategy are also discussed.
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.