2020
Next generation miRNA inhibition using short anti-seed PNAs encapsulated in PLGA nanoparticles
Malik S, Lim J, Slack FJ, Braddock DT, Bahal R. Next generation miRNA inhibition using short anti-seed PNAs encapsulated in PLGA nanoparticles. Journal Of Controlled Release 2020, 327: 406-419. PMID: 32835710, PMCID: PMC7606596, DOI: 10.1016/j.jconrel.2020.08.026.Peer-Reviewed Original ResearchConceptsShort PNA probesPNA probesPLGA nanoparticlesRelease profileNanoparticle formulationNanoparticlesSuperior loadingEfficient transfection efficiencyCationic domainsPotential cancer therapySystemic deliveryTarget proteinsSize distributionProbeTransfection efficiencyMiRNA-155New avenuesPNAUniform distributionProof of conceptCancer therapeuticsCancer therapyReagentsChronic toxicityMicroscopy
2016
In vivo correction of anaemia in β-thalassemic mice by γPNA-mediated gene editing with nanoparticle delivery
Bahal R, Ali McNeer N, Quijano E, Liu Y, Sulkowski P, Turchick A, Lu YC, Bhunia DC, Manna A, Greiner DL, Brehm MA, Cheng CJ, López-Giráldez F, Ricciardi A, Beloor J, Krause DS, Kumar P, Gallagher PG, Braddock DT, Mark Saltzman W, Ly DH, Glazer PM. In vivo correction of anaemia in β-thalassemic mice by γPNA-mediated gene editing with nanoparticle delivery. Nature Communications 2016, 7: 13304. PMID: 27782131, PMCID: PMC5095181, DOI: 10.1038/ncomms13304.Peer-Reviewed Original ResearchConceptsNanoparticle deliveryGene correctionReversal of splenomegalyPeptide nucleic acidLow off-target effectsVivo correctionGenome editingOff-target effectsGene editingHaematopoietic stem cellsNucleic acidsDonor DNAStem cellsΓPNAΒ-thalassaemiaNanoparticlesDeliveryEditingSCF treatmentTriplex formation