Full-length Dysferlin Transfer by the Hyperactive Sleeping Beauty Transposase Restores Dysferlin-deficient Muscle

• Main Authors: Helena Escobar (Author), Verena Schöwel (Author), Simone Spuler (Author), Andreas Marg (Author), Zsuzsanna Izsvák (Author)
• 格式: 圖書
• 出版: Elsevier, 2016-01-01T00:00:00Z.
• 主題: article
• 在線閱讀: Connect to this object online.

Dysferlin-deficient muscular dystrophy is a progressive disease characterized by muscle weakness and wasting for which there is no treatment. It is caused by mutations in DYSF, a large, multiexonic gene that forms a coding sequence of 6.2 kb. Sleeping Beauty (SB) transposon is a nonviral gene transf...