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US 8,409,826 B2Granted
TAT-utrophin as a protein therapy for dystrophinopathies
AssigneeWisconsin Alumni Research Foundation
InventorsJames M. Ervasti, Kevin J. Sonnemann
Life of this patent
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Abstract
Disclosed is a fusion protein including a full-length TAT-utrophin or an anti-dystrophinopathic fragment thereof, a method of treating dystrophinopathies (including Duchenne muscular dystrophy) using the fusion protein, a pharmaceutical composition for treating dystrophinopathies in mammals comprising the fusion protein, and nucleic acid constructs for expressing the fusion protein.
Claims39
An isolated fusion protein comprising:
- a first protein region which is effective to transduce the fusion protein into mammalian muscle cells, operationally linked to;
- a second protein region comprising a full-length utrophin protein or an anti-dystrophinopathic fragment thereof.
An isolated nucleic acid expression construct encoding a fusion protein, the nucleic acid expression construct comprising:
- a first nucleic acid region that encodes a first protein region of the fusion protein, wherein the first protein region is effective to transduce the fusion protein into mammalian muscle cells, operationally linked to;
- a second nucleic acid region that encodes a second protein region of the fusion protein, wherein the second protein region comprises a full-length utrophin protein or an anti-dystrophinopathic fragment thereof;
- wherein the expression construct drives expression of the fusion protein when transformed into a suitable host cell or disposed into a suitable cell-free expression system.
Dependencies are inferred from the claim text.