Pubblicazioni

Time-controlled and muscle-specific CRISPR/Cas9-mediated deletion of CTG-repeat expansion in the DMPK gene

2022 - MOLECULAR THERAPY NUCLEIC ACIDS

Cardinali, B.; Provenzano, C.; Izzo, M.; Voellenkle, C.; Battistini, J.; Strimpakos, G.; Golini, E.; Mandillo, S.; Scavizzi, F.; Raspa, M.; Perfetti, A.; Baci, D.; Lazarevic, D.; Garcia-Manteiga, J. M.; Gourdon, G.; Martelli, F.; Falcone, G.

Hydrophobically Modified let-7b miRNA Enhances Biodistribution to NSCLC and Downregulates HMGA2 InÂ Vivo

2020 - MOLECULAR THERAPY NUCLEIC ACIDS

Segal, Meirav; Biscans, Annabelle; Gilles, Maud-Emmanuelle; Anastasiadou, Eleni; De Luca, Roberto; Lim, Jihoon; Khvorova, Anastasia; Slack, Frank J

CRISPR/Cas9âmediated deletion of CTG expansions recovers normal phenotype in myogenic cells derived from myotonic dystrophy 1 patients

2017 - MOLECULAR THERAPY NUCLEIC ACIDS

Provenzano, Claudia; Cappella, Marisa; Valaperta, Rea; Cardani, Rosanna; Meola, Giovanni; Martelli, Fabio; Cardinali, Beatrice; Falcone, Germana

The Gene Targeting Approach of Small Fragment Homologous Replacement (SFHR) Alters the Expression Patterns of DNA Repair and Cell Cycle Control Genes

2016 - MOLECULAR THERAPY NUCLEIC ACIDS

Pierandrei, Silvia; Andrea, Luchetti; Massimo, Sanchez; Giuseppe, Novelli; Federica, Sangiuolo; Lucarelli, Marco

Pubblicazioni

Time-controlled and muscle-specific CRISPR/Cas9-mediated deletion of CTG-repeat expansion in the DMPK gene

Hydrophobically Modified let-7b miRNA Enhances Biodistribution to NSCLC and Downregulates HMGA2 InÂ Vivo

CRISPR/Cas9âmediated deletion of CTG expansions recovers normal phenotype in myogenic cells derived from myotonic dystrophy 1 patients

The Gene Targeting Approach of Small Fragment Homologous Replacement (SFHR) Alters the Expression Patterns of DNA Repair and Cell Cycle Control Genes

CRISPR/Cas9âmediated deletion of CTG expansions recovers normal phenotype in myogenic cells derived from myotonic dystrophy 1 patients