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.

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

Pubblicazioni

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

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

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