Transposon-mediated Generation of Cellular and Mouse Models of Splicing Mutations to Assess the Efficacy of snRNA-based Therapeutics

• Main Authors: Elena Barbon (Author), Mattia Ferrarese (Author), Laetitia van Wittenberghe (Author), Peggy Sanatine (Author), Giuseppe Ronzitti (Author), Fanny Collaud (Author), Pasqualina Colella (Author), Mirko Pinotti (Author), Federico Mingozzi (Author)
• Format: Book
• Published: Elsevier, 2016-01-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

Disease-causing splicing mutations can be rescued by variants of the U1 small nuclear RNA (U1snRNAs). However, the evaluation of the efficacy and safety of modified U1snRNAs as therapeutic tools is limited by the availability of cellular and animal models specific for a given mutation. Hence, we exp...