Genetic and protein manipulation of betaIG-H3 for the treatment and cure of muscular dystrophies
OwnerRichard G. LeBaron, Jill Whitelock Fergusoninventors
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Abstract
Compositions and methods are disclosed for curing, treating or preventing the onset of Muscular Dystrophies or related neuromuscular diseases, where the compositions include βig-H3, a variant thereof.
Claims20
A composition comprising:
- a therapeutically effective amount of βIG-H3, a βIG-H3 variant, a portion of βIG-H3, a variant of a portion βIG-H3 or mixtures thereof,
- where the amount is sufficient to cure, treat, ameliorate, and/or prevent symptoms of Muscular Dystrophies or related neuromuscular diseases.
A composition comprising:
- an amount of a DNA sequence encoding βIG-H3, a DNA sequence encoding a βIG-H3 variant, a DNA sequence encoding a portion of βIG-H3, a DNA sequence encoding a portion of a βIG-H3 variant, antisense sequences corresponding thereto, or mixtures thereof,
- where the amount is sufficient to cause expression of the sequences in cells of an animal including a human to produce a therapeutically effective amount of encoded polypeptides sufficient to ameliorate, treat, prevent and/or cure Muscular Dystrophies or related neuromuscular diseases.
A plasmid comprising a DNA sequence encoding βIG-H3, a DNA sequence encoding a βIG-H3 variant, a DNA sequence encoding a portion of βIG-H3, a DNA sequence encoding a portion of a βIG-H3 variant, antisense sequences corresponding thereto, or mixtures thereof.
A DNA delivery system comprising a DNA sequence encoding βIG-H3, a DNA sequence encoding a βIG-H3 variant, a DNA sequence encoding a portion of βIG-H3, a DNA sequence encoding a portion of a βIG-H3 variant, antisense sequences corresponding thereto, or mixtures thereof, where the system is selected from the group consisting of a plasmid, a viral DNA delivery system, a liposome DNA delivery system and a mixtures thereof.
A method for treating Muscular Dstrophies or related neuromuscular diseases comprising the step of administering to a patient a therapeutically effective amount of a composition including βIG-H3, a βIG-H3 variant, a portion of βIG-H3, a variant of a portion βIG-H3 or mixtures thereof, where the amount it sufficient to reduce, prevent, cure, and/or treat symptoms associated with Muscular Dstrophies or related neuromuscular diseases.
A method for treating Muscular Dstrophies or related neuromuscular diseases comprising the step of administering to a patient a composition comprising a DNA sequence encoding βIG-H3, a DNA sequence encoding a βIG-H3 variant, a DNA sequence encoding a portion of βIG-H3, a DNA sequence encoding a portion of a βIG-H3 variant, antisense sequences corresponding thereto, or mixtures thereof in an amount sufficient to cause cells in the patient to express a translated polypeptide corresponding to the sequences at a therapeutically effective level to reduce, prevent, cure, ameliorate, and or treat symptoms of Muscular Dstrophies or related neuromuscular diseases.
A method for delaying the onset of Muscular Dystrophies or related neuromuscular diseases comprising administering to a patient a composition comprising βIG-H3, a βIG-H3 variant, a portion of βIG-H3, a variant of a portion βIG-H3 or mixtures thereof according to a prophylactic treatment protocol sufficient to prevent or delay the onset of symptoms of Muscular Dystrophies or related neuromuscular diseases.
Dependencies are inferred from the claim text.