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
2015
Life-Long Transgene Expression in Skeletal Muscle Without Transduction of Satellite Cells Following Embryonic Myogenic Progenitor Transduction by Lentivirus Administered in Utero
Stitelman DH, Brazelton TR, Endo M, Bora A, Traas J, Zoltick PW, Flake AW. Life-Long Transgene Expression in Skeletal Muscle Without Transduction of Satellite Cells Following Embryonic Myogenic Progenitor Transduction by Lentivirus Administered in Utero. Stem Cells And Development 2015, 24: 1878-1887. PMID: 25915576, PMCID: PMC4533031, DOI: 10.1089/scd.2014.0585.Peer-Reviewed Original ResearchMeSH KeywordsAnimalsLentivirusMiceMice, Inbred C57BLMuscle, SkeletalMyogenic Regulatory Factor 5Satellite Cells, Skeletal MuscleTransgenesConceptsSatellite cellsMarker gene expressionMyogenic stem cellsMammalian myogenesisTransgene expressionMuscle progenitorsPrimitive streakMyogenic progenitorsCommon progenitorGene expressionMarker transgeneAdult muscleGene transferTransductionStem cellsCell transductionDermomyotomeProgenitorsSkeletal muscleMuscle fibersSustained transgene expressionExpressionDermatomyotomeCellsRecent evidence