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

• Main Authors: Beatrice Cardinali (Author), Claudia Provenzano (Author), Mariapaola Izzo (Author), Christine Voellenkle (Author), Jonathan Battistini (Author), Georgios Strimpakos (Author), Elisabetta Golini (Author), Silvia Mandillo (Author), Ferdinando Scavizzi (Author), Marcello Raspa (Author), Alessandra Perfetti (Author), Denisa Baci (Author), Dejan Lazarevic (Author), Jose Manuel Garcia-Manteiga (Author), Geneviève Gourdon (Author), Fabio Martelli (Author), Germana Falcone (Author)
• Format: Book
• Published: Elsevier, 2022-03-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

CRISPR/Cas9-mediated therapeutic gene editing is a promising technology for durable treatment of incurable monogenic diseases such as myotonic dystrophies. Gene-editing approaches have been recently applied to in vitro and in vivo models of myotonic dystrophy type 1 (DM1) to delete the pathogenic CT...