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 ResearchMeSH KeywordsAnimalsBrainDiffusion Magnetic Resonance ImagingDisease Models, AnimalFeasibility StudiesFemaleFetusHumansMeningomyelocelePregnancyRatsSpinal CordTretinoinConceptsMagnetic 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) maps
2018
In 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