ArticleThe Journal of clinical investigation2004
Prevention of type 1 diabetes by gene therapy.
Article in The Journal of clinical investigation, 2004. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 24 papers.
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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.
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Who cites it
24 citing papers in PubMed, 86 citations in OpenAlex.
- Disease-modifying pharmacological treatments of type 1 diabetes: Molecular mechanisms, target checkpoints, and possible combinatorial treatments.Pharmacological reviews · 2025Review
- Cell Therapies and Gene Therapy for Diabetes: Current Progress.Current diabetes reviews · 2025Review
- Autoimmune CD8+ T cells in type 1 diabetes: from single-cell RNA sequencing to T-cell receptor redirection.Frontiers in endocrinology · 2024Review
- Programmed cell death in stem cell-based therapy: Mechanisms and clinical applications.World journal of stem cells · 2021Review
- Hematopoietic Stem Cells in Type 1 Diabetes.Frontiers in immunology · 2021Review
- Transplantation of MHC-mismatched mouse embryonic stem cell-derived thymic epithelial progenitors and MHC-matched bone marrow prevents autoimmune diabetes.Stem cell research & therapy · 2019Article
- Recombinant allergens for immunotherapy: state of the art.Current opinion in allergy and clinical immunology · 2019Review
- An Overview on Probiotics as an Alternative Strategy for Prevention and Treatment of Human Diseases.Iranian journal of pharmaceutical research : IJPR · 2019Review
- The site of allergen expression in hematopoietic cells determines the degree and quality of tolerance induced through molecular chimerism.European journal of immunology · 2013Article
- Tolerance to MHC class II disparate allografts through genetic modification of bone marrow.Gene therapy · 2013Article
- Non-myeloablative transplantation of bone marrow expressing self-antigen establishes peripheral tolerance and completely prevents autoimmunity in mice.Gene therapy · 2012Article
- Thymic gene transfer of myelin oligodendrocyte glycoprotein ameliorates the onset but not the progression of autoimmune demyelination.Molecular therapy : the journal of the American Society of Gene Therapy · 2012Article
- Uveitis-associated epitopes of retinal antigens are pathogenic in the humanized mouse model of uveitis and identify autoaggressive T cells.Journal of immunology (Baltimore, Md. : 1950) · 2011Article
- Article
- Induction of transplantation tolerance to fully mismatched cardiac allografts by T cell mediated delivery of alloantigen.Clinical immunology (Orlando, Fla.) · 2010Article
- Approaches in type 1 diabetes research: A status report.International journal of diabetes in developing countries · 2009Article
- The IFN-gamma-induced transcriptional program of the CIITA gene is inhibited by statins.European journal of immunology · 2008Article
- Inhibition of CD26 peptidase activity significantly improves engraftment of retrovirally transduced hematopoietic progenitors.Gene therapy · 2006Article
- Facilitating physiologic self-regeneration: a step beyond islet cell replacement.Pharmaceutical research · 2006Review
- Human gene therapy and imaging in neurological diseases.European journal of nuclear medicine and molecular imaging · 2005Review
Corrections and comments
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Authors and funding
6 authors at 3 institutions in 1 country.
Funding
Abstract
The autoimmune disease type 1 diabetes in humans and NOD mice is determined by multiple genetic factors, among the strongest of which is the inheritance of diabetes-permissive MHC class II alleles associated with susceptibility to disease. Here we examined whether expression of MHC class II alleles associated with resistance to disease could be used to prevent the occurrence of diabetes. Expression of diabetes-resistant MHC class II I-Abeta chain molecules in NOD mice following retroviral transduction of autologous bone marrow hematopoietic stem cells prevented the development of autoreactive T cells by intrathymic deletion and protected the mice from the development of insulitis and diabetes. These data suggest that type 1 diabetes could be prevented in individuals expressing MHC alleles associated with susceptibility to disease by restoration of protective MHC class II expression through genetic engineering of hematopoietic stem cells.
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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.