Santos, José L.Pandita, DeeptiRodrigues, JoãoPêgo, Ana P.Granja, Pedro L.Balian, GaryTomás, Helena2019-06-172019-06-172010-06-07Santos, J. L., Pandita, D., Rodrigues, J., Pêgo, A. P., Granja, P. L., Balian, G., & Tomás, H. (2010). Receptor-mediated gene delivery using PAMAM dendrimers conjugated with peptides recognized by mesenchymal stem cells. Molecular pharmaceutics, 7(3), 763-774.1543-8384http://hdl.handle.net/10400.13/2399As mesenchymalstemcells(MSCs)candifferentiateintomultiplecelltypes,thedeliveryof exogenous genes to this type of cell can be an important tool in tissue regeneration and engineering. HowevertransfectionofMSCsusingnonviralgenedeliveryvectorsisdifficult,thedevelopmentofmore efficientandsafeDNAvehiclesbeingnecessary.Moreover,specifictransfectionofMSCsmayberequired to avoid unwanted side effects in other tissues. In this study, a novel family of gene delivery vectors based on poly(amidoamine) (PAMAM) dendrimers functionalized with peptides displaying high affinity toward MSCs was prepared. The vectors were characterized with respect to their ability to neutralize, bindandcompactplasmidDNA(pDNA).ThecomplexesformedbetweenthevectorsandpDNAwere analyzedconcerningtheirsize, -potential,capacityofbeinginternalizedbycellsandtransfectionefficiency. Thesenewvectorsexhibitedlowcytotoxicity,receptor-mediatedgenedeliveryintoMSCsandtransfection efficiencies superior to those presented by native dendrimers and by partially degraded dendrimers.engPAMAM dendrimersDNAGene deliveryCell recognitionMesenchymal stem cells.Faculdade de Ciências Exatas e da Engenhariajournal article10.1021/mp9002877