Alginate microparticles loaded with basic fibroblast growth factor induce tissue coverage in a rat model of myelomeningocele
Farrelly JS, Bianchi AH, Ricciardi AS, Buzzelli GL, Ahle SL, Freedman-Weiss MR, Luks VL, Saltzman WM, Stitelman DH. Alginate microparticles loaded with basic fibroblast growth factor induce tissue coverage in a rat model of myelomeningocele. Journal Of Pediatric Surgery 2018, 54: 80-85. PMID: 30414695, DOI: 10.1016/j.jpedsurg.2018.10.031.Peer-Reviewed Original ResearchConceptsSoft tissue coverageTissue coverageRat modelTime-dated pregnant ratsFree bFGFGrowth factorInjection of microparticlesBasic fibroblast growth factorFibroblast growth factorPregnant ratsPrenatal treatmentIntraamniotic injectionFetal myelomeningoceleMyelomeningoceleMMC coverageMMC defectsRetinoic acidBFGFIn utero nanoparticle delivery for site-specific genome editing
Ricciardi AS, Bahal R, Farrelly JS, Quijano E, Bianchi AH, Luks VL, Putman R, López-Giráldez F, Coşkun S, Song E, Liu Y, Hsieh WC, Ly DH, Stitelman DH, Glazer PM, Saltzman WM. In utero nanoparticle delivery for site-specific genome editing. Nature Communications 2018, 9: 2481. PMID: 29946143, PMCID: PMC6018676, DOI: 10.1038/s41467-018-04894-2.Peer-Reviewed Original ResearchConceptsSite-specific genome editingReversal of splenomegalyPeptide nucleic acidIntra-amniotic administrationBlood hemoglobin levelsMonogenic disordersNanoparticle deliveryPolymeric nanoparticlesPostnatal elevationGestational ageHemoglobin levelsImproved survivalPediatric morbidityDisease improvementHuman β-thalassemiaReticulocyte countNormal organ developmentMouse modelNormal rangeEarly interventionGenome editingOff-target mutationsPostnatal growthGene editingVersatile method