Restoration of dystrophin expression using the Sleeping Beauty transposon
نویسندگان
چکیده
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.
منابع مشابه
Restoration of dystrophin expression using the Sleeping Beauty transposon – PLOS Currents Muscular Dystrophy
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 ΔR4R23/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...
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عنوان ژورنال:
دوره 3 شماره
صفحات -
تاریخ انتشار 2011