Development of a novel PTD-mediated IVT-mRNA delivery platform for potential protein replacement therapy of metabolic/genetic disorders
Αndroulla N. Miliotou, Ioannis S. Pappas, George Spyroulias, Efthimia Vlachaki, Asterios S. Tsiftsoglou, Ioannis S. Vizirianakis, Lefkothea C. Papadopoulou
Abstract
was transduced and translated in bone marrow cells, derived from three β-thalassemic patients. The transducibility and the structural stability of the PDT-IVT-mRNAs, in both cases, were confirmed at the RNA and protein levels. We propose that our novel delivery platform could be clinically applicable as a protein therapy for metabolic/genetic disorders.
Topics & Concepts
Computational biologyMedicineBioinformaticsMessenger RNABiologyGeneticsGeneRNA Interference and Gene DeliveryVirus-based gene therapy researchViral Infectious Diseases and Gene Expression in Insects