2003
Sequence-specific modification of genomic DNA by small DNA fragments
Gruenert DC, Bruscia E, Novelli G, Colosimo A, Dallapiccola B, Sangiuolo F, Goncz KK. Sequence-specific modification of genomic DNA by small DNA fragments. Journal Of Clinical Investigation 2003, 112: 637-641. PMID: 12952908, PMCID: PMC182219, DOI: 10.1172/jci19773.Peer-Reviewed Original ResearchConceptsSmall fragment homologous replacementSequence-specific modificationSmall DNA fragmentsGenomic DNADNA fragmentsEndogenous genomic DNADuchenne muscular dystrophyTherapeutic modalitiesCystic fibrosisHomologous replacementGenomic editingMuscular dystrophyGenetic lociMouse cellsUnderlying mechanismPhenotypic analysisSpecific modificationsDNA
2002
In vitrocorrection of cystic fibrosis epithelial cell lines by small fragment homologous replacement (SFHR) technique
Sangiuolo F, Bruscia E, Serafino A, Nardone AM, Bonifazi E, Lais M, Gruenert DC, Novelli G. In vitrocorrection of cystic fibrosis epithelial cell lines by small fragment homologous replacement (SFHR) technique. BMC Medical Genomics 2002, 3: 8. PMID: 12243649, PMCID: PMC130050, DOI: 10.1186/1471-2350-3-8.Peer-Reviewed Original ResearchCell linesMembrane fusion pathwayCystic fibrosis epithelial cell lineDNA ratioSequence-based analysisCultured cell linesLipid/DNA ratioNormal airway epithelial cellsHomologous replacementMammalian cellsEndocytic pathwayLipid/DNA complexesEpithelial cell lineGene targetingFusion pathwayCellular factorsGenetic targetsCellular DNASmall fragmentsSpecific cell linesDNA complexesCellular processingSpecific mutationsDNASFHR