Effective gene therapy of Stargardt disease with PEG-ECO/pGRK1-ABCA4-S/MAR nanoparticles
Da Sun, Wenyu Sun, Songqi Gao, Jonathan Lehrer, Amirreza Naderi, Cheng Wei, Sang-Joon Lee, Andrew L. Schilb, Josef H. Scheidt, Ryan Hall, Elias I. Traboulsi, Krzysztof Palczewski, Zheng‐Rong Lu
Abstract
nanoparticles are a promising non-viral mediated gene therapy modality for STGD type 1 (STGD1).
Topics & Concepts
ABCA4Stargardt diseasePEG ratioGenetic enhancementNanoparticleMedicineNanotechnologyChemistryMaterials scienceGeneBiochemistryBusinessFinancePhenotypeRetinal Development and DisordersRNA Interference and Gene DeliveryVirus-based gene therapy research