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

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

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...

Full description

Bibliographic Details
Main Authors: Elena Barbon, Mattia Ferrarese, Laetitia van Wittenberghe, Peggy Sanatine, Giuseppe Ronzitti, Fanny Collaud, Pasqualina Colella, Mirko Pinotti, Federico Mingozzi
Format: Article
Language:English
Published: Elsevier 2016-01-01
Series:Molecular Therapy: Nucleic Acids
Subjects:
coagulation factor IX
Sleeping Beauty
SB100X
splicing
Online Access:http://www.sciencedirect.com/science/article/pii/S2162253117301178

Internet

http://www.sciencedirect.com/science/article/pii/S2162253117301178

Similar Items