2021
Diffusion weighted imaging as a biomarker of retinoic acid induced myelomeningocele
Maassel N, Farrelly J, Coman D, Freedman-Weiss M, Ahle S, Ullrich S, Yung N, Hyder F, Stitelman D. Diffusion weighted imaging as a biomarker of retinoic acid induced myelomeningocele. PLOS ONE 2021, 16: e0253583. PMID: 34191842, PMCID: PMC8244849, DOI: 10.1371/journal.pone.0253583.Peer-Reviewed Original ResearchConceptsMagnetic resonance imaging techniquesSpinal cordRat fetusesRetinoic acidCommon congenital anomalyComplementary imaging biomarkerLack of skinNeural tube defectsTrans retinoic acidBony coveringRat damsCongenital anomaliesRat modelExperimental therapiesHistopathologic analysisSevere formBrain volumeMyelomeningoceleImaging biomarkersDisease severityMMC defectsTube defectsIncomplete closureFetusesFractional anisotropy (FA) mapsEngineering alginate microparticles for optimized accumulation in fetal rat myelomeningocele
Freedman-Weiss MR, Wu D, Maassel N, Ullrich SJ, Ahle SL, Roberts K, Farrelly JS, Buzzelli G, Saltzman WM, Stitelman DH. Engineering alginate microparticles for optimized accumulation in fetal rat myelomeningocele. Journal Of Pediatric Surgery 2021, 57: 544-550. PMID: 33933264, DOI: 10.1016/j.jpedsurg.2021.03.060.Peer-Reviewed Original Research
2018
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