Trial reportBlood2007
Gene therapy improves immune function in preadolescents with X-linked severe combined immunodeficiency.
Trial report in Blood, 2007. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 43 papers, 1 of them a synthesis that pooled it.
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
43 citing papers in PubMed, 1 synthesis or guideline pooled it, 99 citations in OpenAlex.
- A systematic review and meta-analysis of gene therapy with hematopoietic stem and progenitor cells for monogenic disorders.Nature communications · 2022Pooled it
- Lentiviral hematopoietic stem cell gene therapy for X-linked severe combined immunodeficiency.Science translational medicine · 2016Trial
- Parental Views on the Psychosocial Impact of False-Positive Results Following Newborn Screening for Severe Combined Immunodeficiency in England.International journal of neonatal screening · 2026Article
- The Transfer of the Hepatocyte Growth Factor Gene by Macrophages Ameliorates the Progression of Peritoneal Fibrosis in Mice.International journal of molecular sciences · 2023Article
- Gene therapy and genome editing for primary immunodeficiency diseases.Genes & diseases · 2020Review
- Immune Reconstitution After Gene Therapy Approaches in Patients With X-Linked Severe Combined Immunodeficiency Disease.Frontiers in immunology · 2020Review
- Gene therapy targeting haematopoietic stem cells for inherited diseases: progress and challenges.Nature reviews. Drug discovery · 2019Review
- Genetic Engineering and Manufacturing of Hematopoietic Stem Cells.Molecular therapy. Methods & clinical development · 2017Review
- Evolving Gene Therapy in Primary Immunodeficiency.Molecular therapy : the journal of the American Society of Gene Therapy · 2017Review
- Current status of ex vivo gene therapy for hematological disorders: a review of clinical trials in Japan around the world.International journal of hematology · 2016Review
- A novel intranuclear RNA vector system for long-term stem cell modification.Gene therapy · 2016Article
- Insulators to Improve the Safety of Retroviral Vectors for HIV Gene Therapy.Biomedicines · 2016Article
- Gene Therapy for X-Linked Severe Combined Immunodeficiency: Where Do We Stand?Human gene therapy · 2016Review
- Outcomes in two Japanese adenosine deaminase-deficiency patients treated by stem cell gene therapy with no cytoreductive conditioning.Journal of clinical immunology · 2015Article
- Assessment of Immature Platelet Fraction in the Diagnosis of Wiskott-Aldrich Syndrome.Frontiers in pediatrics · 2015Article
- Lentiviral vectors for the treatment of primary immunodeficiencies.Journal of inherited metabolic disease · 2014Review
- Concise review: lessons learned from clinical trials of gene therapy in monogenic immunodeficiency diseases.Stem cells translational medicine · 2014Review
- Human syndromes of immunodeficiency and dysregulation are characterized by distinct defects in T-cell receptor repertoire development.The Journal of allergy and clinical immunology · 2014Article
- Establishing diagnostic criteria for severe combined immunodeficiency disease (SCID), leaky SCID, and Omenn syndrome: the Primary Immune Deficiency Treatment Consortium experience.The Journal of allergy and clinical immunology · 2014Article
- Gene transfer into hematopoietic stem cells as treatment for primary immunodeficiency diseases.International journal of hematology · 2014Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
13 authors at 3 institutions in 1 country.
Funding
Abstract
Retroviral gene therapy can restore immunity to infants with X-linked severe combined immunodeficiency (XSCID) caused by mutations in the IL2RG gene encoding the common gamma chain (gammac) of receptors for interleukins 2 (IL-2), -4, -7, -9, -15, and -21. We investigated the safety and efficacy of gene therapy as salvage treatment for older XSCID children with inadequate immune reconstitution despite prior bone marrow transplant from a parent. Subjects received retrovirus-transduced autologous peripherally mobilized CD34(+) hematopoietic cells. T-cell function significantly improved in the youngest subject (age 10 years), and multilineage retroviral marking occurred in all 3 children.
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.