Vio, ValentinaRiveros, Ana L.Tapia-Bustos, AndreaLespay-Rebolledo, CarolynePérez-Lobos, RonaldMuñoz, LuisPismante, PaolaMorales, PaolaAraya, EyleenHassan, NataliaHerrera-Marschitz, MarioKogan, Marcelo J.2022-09-092022-09-092018International Journal of Nanomedicine Volume 13, Pages 6839 - 6854 201811769114https://repositorio.unab.cl/xmlui/handle/ria/23820Indexación ScopusBackground: Perinatal asphyxia interferes with neonatal development, resulting in long-term deficits associated with systemic and neurological diseases. Despite the important role of poly (ADP-ribose) polymerase 1 (PARP-1) in the regulation of gene expression and DNA repair, overactivation of PARP-1 in asphyxia-exposed animals worsens the ATP-dependent energetic crisis. Inhibition of PARP-1 offers a therapeutic strategy for diminishing the effects of perinatal asphyxia. Methods: We designed a nanosystem that incorporates a specific siRNA for PARP-1 knockdown. The siRNA was complexed with gold nanorods (AuNR) conjugated to the peptide CLPFFD for brain targeting. Results: The siRNA was efficiently delivered into PC12 cells, resulting in gene silencing. The complex was administered intraperitoneally in vivo to asphyxia-exposed rat pups, and the ability of the AuNR-CLPFFD/siRNA complex to reach the brain was demonstrated. Conclusion: The combination of a nanosystem for delivery and a specific siRNA for gene silencing resulted in effective inhibition of PARP-1 in vivo. © 2018 Vio et al.enADP-ribosylationPoly ADP-ribose GlycohydrolaseDNA DamageGold nanorodsIn vivo administrationNeonatal hypoxiasiRNA deliveryArtículoCC BY-NC 4.010.2147/IJN.S175076