Microdystrophin peptides and methods for treating muscular dystrophy using the same
AssigneeThe Curators of the University of Missouri
InventorsYi Lai, Junling Zhao, Yongping Yue, Dongsheng Duan
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Abstract
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.
Claims21
A therapeutic composition comprising:
- a microdystrophin peptide having an amino acid sequence which comprises dystrophin spectrin-like repeats 16 and 17 (R16/R17) (SEQ ID NO:7);
- and
- a delivery vehicle.
A therapeutic composition comprising:
- an amino acid sequence motif which comprises RFHYDIKIFN (SEQ ID NO:46);
- and
- a delivery vehicle.
A method of treating DMD, BMD or XLDC comprising administering a therapeutic amount of a therapeutic composition to a subject having DMD or BMD, wherein the therapeutic composition comprises
- an amino acid sequence motif RFHYDIKIFN (SEQ ID NO:46);
- and
- a delivery vehicle.
Dependencies are inferred from the claim text.