Microdystrophin peptides and methods for treating muscular dystrophy using the same

摘要

According to the embodiments described herein, a series of biological materials for treatment/therapy of DMD and/or BMD through the recovery of sarcolemmal nNOS is provided. The biological material comprises the complete dystrophin repeats R16 and R17 or certain domains, sections, or fragments of the dystrophin repeats R16 and R17. In some aspects, such domains, sections, or fragments may be selected from sequence motifs including dystrophin R17 α1 helix, α2 and α3 helices of both R16 and R17, or a combination thereof.

主权项

1. A therapeutic composition comprising:
a microdystrophin peptide having an amino acid sequence which consists essentially of dystrophin spectrin-like repeats 16 and 17 (R16/R17) (SEQ ID NO:7); and a delivery vehicle, wherein the delivery vehicle is a cell-penetrating peptide.