Granted Patent
B2
US 12,421,285 · App. 16/593,840 · Granted Sep 23, 2025
Micro-dystrophins and related methods of use
Inventors:
Jeffrey S. Chamberlain (Seattle, WA); Julian Ramos (Seattle, WA); Stephen D. Hauschka (Seattle, WA)
Assignee:
University of Washington
C07K14/4708A61K48/0058A61P21/00C07H21/04C07K14/4707C07K14/4716C12N15/85C12N15/86A61K38/00C07K2319/30C12N2750/14143C12N2830/008
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Abstract
Nucleotide sequences including a micro-dystrophin gene are provided. The micro-dystrophin genes may be operatively linked to a regulatory cassette. Methods of treating a subject having, or at risk of developing, muscular dystrophy, sarcopenia, heart disease, or cachexia are also provided. The methods may include administering a pharmaceutical composition including the micro-dystrophin gene and a delivery vehicle to a subject. Further, the methods may include administering the pharmaceutical composition a subject having Duchenne muscular dystrophy or Becker muscular dystrophy.
Claims (8)
1. A method for treating a subject having muscular dystrophy or prophylactically treating a subject at risk of developing muscular dystrophy, comprising:
administering to the subject a therapeutically effective amount of a pharmaceutical composition comprising a micro-dystrophin gene operably coupled to a regulatory cassette;
wherein the micro-dystrophin gene encodes a protein having at least 91% sequence identity to the amino acid sequence of SEQ ID NO: 4.
2. The method of claim 1 , wherein the regulatory cassette is selected from the group consisting of a CK8 promoter and a cardiac troponin T (cTnT) promoter.
3. The method of claim 1 , wherein the regulatory cassette is a CK8 promoter, and wherein the CK8 promoter has at least 80% sequence identity to the nucleic acid sequence of SEQ ID NO: 19.
4. The method of claim 1 , wherein the regulatory cassette is a cTnT promoter, and wherein the cTnT promoter has at least 80% sequence identity to the nucleic acid sequence of SEQ ID NO:1.
5. The method of claim 1 , wherein the regulatory cassette is configured to express the micro-dystrophin gene such that a level of expression of the micro-dystrophin gene is at least 100-fold higher in striated muscle cells than the level of expression of the micro-dystrophin gene in non-muscle cells.
6. The method of claim 1 , wherein the micro-dystrophin gene encodes a protein having the amino acid sequence of SEQ ID NO: 4.
Assignments (1)
ASSIGNMENT OF ASSIGNOR'S INTEREST
Recorded Jan 7, 2020
From: CHAMBERLAIN, JEFFREY S; RAMOS, JULIAN; HAUSCHKA, STEPHEN D
To: UNIVERSITY OF WASHINGTON
Reel/Frame 051439/0037 →
Continuity (3)
Division
15541870
Provisional Application
62104537
· Jan 16, 2015
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