Development of a beta cell-specific expression control element for rAAV

Consortia: CTAR

Chai S, Kim Y, Galivo F, Dorrell C, Wakefield L, Posey J, Ackermann AM, Kaestner KH, Hebrok M, Grompe M.
Hum Gene Ther. 2022 Mar 17. doi: 10.1089/hum.2021.219. Online ahead of print. PMID:35297680

All Exploring Computational Data Amplification and Imputation for the Discovery of Type 1 Diabetes (T1D) Biomarkers from Limited Human Datasets The type 1 diabetes gene TYK2 regulates β-cell development and its responses to interferon-α Pro-Inflammatory Cytokines Promote the Transcription of Circular RNAs in Human Pancreatic β Cells Mouse models and human islet transplantation sites for intravital imaging Direct Reprogramming of Different Cell Lineages into Pancreatic β-Like Cells Yo-Yo Dieting: Mixed Messages for β-Cell Plasticity Stem cell-based multi-tissue platforms to model human autoimmune diabetes Understanding islet dysfunction in type 2 diabetes through multidimensional pancreatic phenotyping: The Human Pancreas Analysis Program Aging compromises human islet beta cell function and identity by decreasing transcription factor activity and inducing ER stress A human iPSC-array-based GWAS identifies a virus susceptibility locus in the NDUFA4 gene and functional variants Fluorescence imaging of beta cell primary cilia Recognition of mRNA Splice Variant and Secretory Granule Epitopes by CD4+ T Cells in Type 1 Diabetes Identification of T cell antigens in the 21st century, as difficult as ever Editorial: alternative antigen processing and presentation in immune disorders Islet primary cilia motility controls insulin secretion d-Amino Acids and Classical Neurotransmitters in Healthy and Type 2 Diabetes-Affected Human Pancreatic Islets of Langerhans Transcription and splicing regulation by NLRC5 shape the interferon response in human pancreatic β cells Functional architecture of pancreatic islets identifies a population of first responder cells that drive the first-phase calcium response Genetic activation of glucokinase in a minority of pancreatic beta cells causes hypoglycemia in mice Ca2+ Oscillations, Waves, and Networks in Islets from Human Donors With and Without Type 2 Diabetes 3D chromatin maps of the human pancreas reveal lineage-specific regulatory architecture of T2D risk Enhanced development of functional human NK cells in NOD-scid-IL2rg(null) mice expressing human IL15 Carbonyl Posttranslational Modification Associated With Early-Onset Type 1 Diabetes Autoimmunity IL-2 receptor engineering enhances regulatory T cell function suppressed by calcineurin inhibitor Precision medicine in type 1 diabetes Alpha cell TXNIP deletion improves diabetes-associated hyperglycemia and hyperglucagonemia Every islet matters: improving the impact of human islet research Adaptation to chronic ER stress enforces pancreatic β-cell plasticity Predicting Misdiagnosed Adult-onset Type 1 Diabetes Using Machine Learning EZH1 repression generates mature iPSC-derived CAR T cells with enhanced antitumor activity The β-Cell in Type 1 Diabetes Pathogenesis: A Victim of Circumstances or an Instigator of Tragic Events? The Yin and Yang of Modulating β-Cell DNA Damage Response and Functional Mass Cilia Action in Islets: Lessons From Mouse Models Integrating Additive Manufacturing Techniques to Improve Cell-Based Implants for the Treatment of Type 1 Diabetes Characterizing cis-regulatory elements using single-cell epigenomics Extracellular vesicles in β cell biology: Role of lipids in vesicle biogenesis, cargo, and intercellular signaling Type I but Not Type II Calreticulin Mutations Activate the IRE1α/XBP1 Pathway of the Unfolded Protein Response to Drive Myeloproliferative Neoplasms CRISPR screening uncovers a central requirement for HHEX in pancreatic lineage commitment and plasticity restriction Mitofusins Mfn1 and Mfn2 Are Required to Preserve Glucose- but Not Incretin-Stimulated β-Cell Connectivity and Insulin Secretion Proinflammatory signaling in islet β cells propagates invasion of pathogenic immune cells in autoimmune diabetes TCF-1 promotes chromatin interactions across topologically associating domains in T cell progenitors Increasing insulin measurement throughput by fluorescence anisotropy imaging immunoassays α Cell dysfunction in islets from nondiabetic, glutamic acid decarboxylase autoantibody-positive individuals Islet cell replacement and transplantation immunology in a mouse strain with inducible diabetes LGR4, a G Protein-Coupled Receptor With a Systemic Role: From Development to Metabolic Regulation Human CD4(+)CD25(+)CD226(-) Tregs Demonstrate Increased Purity, Lineage Stability, and Suppressive Capacity Versus CD4(+)CD25(+)CD127(lo/-) Tregs for Adoptive Cell Therapy T1D patient-derived hematopoietic stem cells are programmed to generate Tph, Tfh, and autoimmunity-associated B cell subsets in human immune system mice An intrinsically disordered protein region encoded by the human disease gene CLEC16A regulates mitophagy Polycomb Repressive Complexes: Shaping Pancreatic Beta-Cell Destiny in Development and Metabolic Disease The Foundation for Engineering a Pancreatic Islet Niche
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