Patent ID: 7510867

Claim:
An isolated nucleotide sequence comprising a dystrophin minigene encoding a protein consisting of: (a) a N-terminal domain; (b) not less than five nor more than six rod repeats; (c) an H1 domain of a dystrophin protein and an H4 domain of the dystrophin protein; and (d) a cysteine-rich domain, wherein: the N-terminal domain is selected from a group consisting of a N-terminal domain of the dystrophin protein, a modified N-terminal domain of the dystrophin protein, and a N-terminal domain of a utrophin protein; the rod repeats are selected from a group consisting of rod repeats in the dystrophin protein, rod repeats in the utrophin protein, and rod repeats in a spectrin protein; the cysteine-rich domain is the cysteine-rich domain of the dystrophin protein or the utrophin protein, and wherein the dystrophin minigene is capable of ameliorating dystrophic pathology when expressed in a dystrophic muscle.