Formulation and efficacy of ECO/pRHO-ABCA4-SV40 nanoparticles for nonviral gene therapy of Stargardt disease in a mouse model
Da Sun, Wenyu Sun, Songqi Gao, Cheng Wei, Amirreza Naderi, Andrew L. Schilb, Josef H. Scheidt, Sang Joon Lee, Timothy S. Kern, Krzysztof Palczewski, Zheng‐Rong Lu
Topics & Concepts
ABCA4Genetic enhancementStargardt diseaseTransfectionSorbitolBiologyIn vivoMolecular biologyPlasmidIn vitroGene deliveryGeneChemistryBiochemistryGeneticsPhenotypeRNA Interference and Gene DeliveryAdvanced biosensing and bioanalysis techniquesVirus-based gene therapy research