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

• मुख्य लेखकों: Helena Escobar (लेखक), Verena Schöwel (लेखक), Simone Spuler (लेखक), Andreas Marg (लेखक), Zsuzsanna Izsvák (लेखक)
• स्वरूप: पुस्तक
• प्रकाशित: Elsevier, 2016-01-01T00:00:00Z.
• विषय: article
• ऑनलाइन पहुंच: 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...