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agent
Timed GDNF gene therapy using an immune-evasive gene switch promotes long distance axon regeneration.Autoimmunity against a defective ribosomal insulin gene product in type 1 diabetesDegenerated Cones in Cultured Human Retinas Can Successfully Be Optogenetically Reactivatedβ-Cell Stress Shapes CTL Immune Recognition of Preproinsulin Signal Peptide by Posttranscriptional Regulation of Endoplasmic Reticulum Aminopeptidase 1Intronic SMCHD1 variants in FSHDCharacterization of an immune-evading doxycycline-inducible lentiviral vector for gene therapy in the spinal cordProteasomal Degradation of Proinsulin Requires Derlin-2, HRD1 and p97Lentiviral vector-mediated reporter gene expression in avulsed spinal ventral root is short-term, but is prolonged using an immune "stealth" transgeneHuman iPSC-Derived Retinas Recapitulate the Fetal CRB1 CRB2 Complex Formation and Demonstrate that Photoreceptors and Müller Glia Are Targets of AAV5Highly efficient ex vivo lentiviral transduction of primary human pancreatic exocrine cellsSingle-Cell Transcriptomics Links Loss of Human Pancreatic β-Cell Identity to ER StressBioluminescent reporter assay for monitoring ER stress in human beta cells
author
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Rob C Hoeben