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AssigneeThe Curators of the University of Missouri
InventorsDongsheng Duan, Yi Lai, Junling Zhao, Yongping Yue
Estimating the term…
Synthetic nucleic acids encoding mini and microdystrophin genes comprising the membrane binding motifs or domains of the R10-R11-R12 region are provided. Also provided are vectors, host cells, and related methods of using the same to treat a subject suffering from Duchenne muscular dystrophy (DMD), Becker muscular dystrophy (BMD) or X-linked dilated cardiomyopathy (XLDC), or for ameliorating one or more adverse effects of DMD, BMD, or XLDC. Also provided are a fusion protein comprising a nNOS binding domain of dystrophin R16-R17 that is operably linked to a syntrophin PDZ domain and synthetic nucleic acids comprising the same that can be used to treat subjects with diseases characterized by loss of sarcolemmal neuronal nitric oxide synthase (nNOS) activity.
1-50. (canceled)
A synthetic nucleic acid molecule comprising a sequence encoding a fusion protein comprising a nNOS binding domain of dystrophin R16-R17 that is operably linked to a syntrophin PDZ domain.
The synthetic nucleic acid molecule of claim 51, wherein alpha-helix 2 and alpha-helix 3 of the R16 domain is present and alpha-helix 1, alpha-helix 2, and alpha-helix 3 of the R17 domain is present in the fusion protein.
54-55. (canceled)
(canceled)
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A fusion protein comprising dystrophin nNOS binding domain of R16-R17 that is operably linked to a syntrophin PDZ domain.
The fusion protein of claim 71, wherein the n-terminal alpha helix of the R16 domain (SEQ ID NO:
Dependencies are inferred from the claim text.