ArticleMolecular therapy : the journal of the American Society of Gene Therapy2013
Genetically engineered human islets protected from CD8-mediated autoimmune destruction in vivo.
Article in Molecular therapy : the journal of the American Society of Gene Therapy, 2013. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 19 papers.
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Who cites it
19 citing papers in PubMed, 29 citations in OpenAlex.
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- Immune Shielding of Human Heart Valves: A Proof-of-Concept Study of HLA-Targeting Therapy for Transplantations.JACC. Basic to translational science · 2024Article
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- From Disease and Patient Heterogeneity to Precision Medicine in Type 1 Diabetes.Frontiers in medicine · 2022Review
- Preclinical Models to Evaluate the Human Response to Autoantigen and Antigen-Specific Immunotherapy in Human Type 1 Diabetes.Frontiers in endocrinology · 2022Review
- Single-Cell Transcriptomics Links Loss of Human Pancreatic β-Cell Identity to ER Stress.Cells · 2021Article
- Review
- Externally-Controlled Systems for Immunotherapy: From Bench to Bedside.Frontiers in immunology · 2020Review
- Highly efficient ex vivo lentiviral transduction of primary human pancreatic exocrine cells.Scientific reports · 2019Article
- Bioluminescent reporter assay for monitoring ER stress in human beta cells.Scientific reports · 2018Article
- Human beta cell mass and function in diabetes: Recent advances in knowledge and technologies to understand disease pathogenesis.Molecular metabolism · 2017Review
- Herpesvirus microRNAs for use in gene therapy immune-evasion strategies.Gene therapy · 2017Review
- The elusive role of B lymphocytes and islet autoantibodies in (human) type 1 diabetes.Diabetologia · 2017Article
- Human islets and dendritic cells generate post-translationally modified islet autoantigens.Clinical and experimental immunology · 2016Article
- Age-Dependent Pancreatic Gene Regulation Reveals Mechanisms Governing Human β Cell Function.Cell metabolism · 2016Article
- Efficient Gene Transduction of Dispersed Islet Cells in Culture Using Fiber-Modified Adenoviral Vectors.Cell medicine · 2015Article
- Toward beta cell replacement for diabetes.The EMBO journal · 2015Review
- Effect and mechanisms of human Wharton's jelly-derived mesenchymal stem cells on type 1 diabetes in NOD model.Endocrine · 2015Article
Corrections and comments
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Authors and funding
12 authors at 3 institutions in 2 countries.
Funding
Abstract
Islet transplantation is a promising therapy for type 1 diabetes, but graft function and survival are compromised by recurrent islet autoimmunity. Immunoprotection of islets will be required to improve clinical outcome. We engineered human β cells to express herpesvirus-encoded immune-evasion proteins, "immunevasins." The capacity of immunevasins to protect β cells from autoreactive T-cell killing was evaluated in vitro and in vivo in humanized mice. Lentiviral vectors were used for efficient genetic modification of primary human β cells without impairing their function. Using a novel β-cell-specific reporter gene assay, we show that autoreactive cytotoxic CD8(+) T-cell clones isolated from patients with recent onset diabetes selectively destroyed human β cells, and that coexpression of the human cytomegalovirus-encoded US2 protein and serine proteinase inhibitor 9 offers highly efficient protection in vitro. Moreover, coimplantation of these genetically modified pseudoislets with β-cell-specific cytotoxic T cells into immunodeficient mice achieves preserved human insulin production and C-peptide secretion. Collectively, our data provide proof of concept that human β cells can be efficiently genetically modified to provide protection from killing mediated by autoreactive T cells and retain their function in vitro and in vivo.
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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.