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Updated: May 25, 2026
CRISPR/Cas9 Technology in Restoring Dystrophin Expression in iPSC-Derived Muscle Progenitors
Published on: September 14, 2019
Restoration of dystrophin expression using the Sleeping Beauty transposon
Sofia Muses1, Jennifer E Morgan, Dominic J Wells
1Department of Comparative and Biomedical Sciences, The Royal Veterinary College, Royal College Street, London NW1 0TU, U.K. and The Dubowitz Neuromuscular Centre UCL Institute of Child Health 30 Guilford Street London WC1N 1EH United Kingdom.
Abstract:
The Sleeping beauty (SB) system is a non-viral DNA based vector that has been used to stably integrate therapeutic genes into disease models. Here we report the SB system is capable of stably integrating the ΔR4-R23/CTΔ micro-dystrophin gene into a conditionally immortal dystrophin deficient muscle cell-line, H2K SF1, a murine cell model for Duchenne muscular dystrophy. Genetically corrected H2K SF1 cells retained their myogenic properties in vitro. Moreover, upon transplantation ΔR4-R23/CTΔ micro-dystrophin expression was detected within mdx nu/nu mice. Our data suggests the SB system is an effective way of stably integrating therapeutic genes into myogenic cells.
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