Granted Patent
B2
US 12,285,497 · App. 18/046,740 · Granted Apr 29, 2025
Self-complementary adeno-associated virus vector and its use in treatment of muscular dystrophy
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Abstract
Described herein are methods of treating muscular dystrophy comprising administering a self complementary recombinant AAV (rAAV) ScAAVrh74.MIHCK7.h8GCB vector, methods of expressing beta-sarcoglycan gene in a patient, pharmaceutical compositions comprising the rAAV, and methods of generating the rAAV.
Claims (11)
1. A polynucleotide sequence comprising a first nucleotide sequence at least 95% identical to the nucleotide sequence of SEQ ID NO: 1, wherein SEQ ID NO: 1 comprises a second nucleotide sequence encoding a β-sarcoglycan protein.
2. The polynucleotide sequence of claim 1 , wherein the first nucleotide sequence comprises the nucleotide sequence of SEQ ID NO: 1.
3. A composition comprising a recombinant AAV comprising the polynucleotide sequence of claim 1 .
4. A method of treating muscular dystrophy in a subject in need thereof comprising administering to the subject a therapeutically effective amount of a recombinant AAV comprising the polynucleotide sequence of claim 1 .
5. The method of claim 4 , wherein the subject is suffering from limb-girdle muscular dystrophy.
6. The method of claim 4 , wherein the recombinant AAV is administered systemically.
7. The method of claim 4 , wherein the recombinant AAV is administered at i) a dosage of 7.41×10 13 vg/kg measured by qPCR using linear reference plasmid or its equivalent dosage of 2×10 14 vg/kg measured by qPCR using supercoiled reference plasmid or ii) a dosage of 1.85×10 13 vg/kg measured by qPCR using linear reference plasmid or its equivalent dosage of 5×10 13 vg/kg measured by qPCR using supercoiled reference plasmid.
8. The method of claim 7 , wherein the recombinant AAV is administered intravenously.
9. A method of increasing muscle mass in a subject suffering from muscular dystrophy comprising administering to the subject a therapeutically effective amount of a recombinant AAV comprising the polynucleotide sequence of claim 1 .
10. A method of treating β-sarcoglycanopathy in a subject comprising administering to the subject a therapeutically effective amount of a recombinant AAV comprising the polynucleotide sequence of claim 1 .
11. A method of increasing beta-sarcoglycan positive fibers and/or decreasing CK levels in a subject's muscle tissue comprising administering to the subject a therapeutically effective amount of a recombinant AAV comprising the polynucleotide sequence of claim 1 .
Assignments (1)
ASSIGNMENT OF ASSIGNOR'S INTEREST
Recorded May 13, 2024
From: RODINO-KLAPAC, LOUISE; MENDELL, JERRY R.
To: RESEARCH INSTITUTE AT NATIONWIDE CHILDREN'S HOSPITAL
Reel/Frame 067390/0580 →
Continuity (2)
Provisional Application
63256368
· Oct 15, 2021
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