2023
AAV-mediated delivery of a Sleeping Beauty transposon and an mRNA-encoded transposase for the engineering of therapeutic immune cells
Ye L, Lam S, Yang L, Suzuki K, Zou Y, Lin Q, Zhang Y, Clark P, Peng L, Chen S. AAV-mediated delivery of a Sleeping Beauty transposon and an mRNA-encoded transposase for the engineering of therapeutic immune cells. Nature Biomedical Engineering 2023, 8: 132-148. PMID: 37430157, PMCID: PMC11320892, DOI: 10.1038/s41551-023-01058-6.Peer-Reviewed Original ResearchGene delivery systemsAdeno-associated virusTransgene expressionHigh transgene expressionSleeping Beauty (SB) transposonTransgene deliverySleeping Beauty transposasePluripotent stem cellsSB transposonLentiviral vectorsGenomic integrationEngineering cellsBeauty transposonMinicircle DNATransposon DNABeauty transposaseCell viabilityPermanent integrationStem cellsElectroporation of plasmidsTransgeneChimeric antigen receptorDeliveryElectroporationEngineering
2015
Hydroxyl PAMAM dendrimer-based gene vectors for transgene delivery to human retinal pigment epithelial cells
Mastorakos P, Kambhampati S, Mishra M, Wu T, Song E, Hanes J, Kannan R. Hydroxyl PAMAM dendrimer-based gene vectors for transgene delivery to human retinal pigment epithelial cells. Nanoscale 2015, 7: 3845-3856. PMID: 25213606, PMCID: PMC4797994, DOI: 10.1039/c4nr04284k.Peer-Reviewed Original ResearchConceptsTransgene deliveryGene vectorsNon-viral gene vectorsHigh-level transgene expressionNon-viral gene deliveryGene delivery platformGene-based therapeuticsOcular gene therapyGene deliveryColloidal stabilityNumerous blinding disordersGene therapyRetinal pigment epitheliumTransgene expressionHuman RPE cellsDNA complexationVector surfacePolyamidoamine dendrimersCell uptakePEGylated dendrimersTriamcinolone acetonideImproved stabilityRPE cellsAmine groupsInherent drawbacks
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