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US 12,391,928 B2Granted
Recombinant adeno-associated virus products and methods for treating limb girdle muscular dystrophy 2A
AssigneeResearch Institute At Nationwide Children's Hospital
InventorZarife Sahenk
Life of this patent
Estimating the term…
Abstract
Products and methods for treating limb girdle muscular dystrophy 2A are provided. In the methods, recombinant adeno-associated viruses deliver DNA encoding a protein with calpain 3 activity.
Claims4
A recombinant adeno-associated virus (rAAV) comprising a polynucleotide which comprises nucleotides 1 to 3977 of SEQ ID NO:
- 1, wherein the rAAV comprises an AAV-8, an AAV-9, or an AAV rh.74 capsid protein, or a variant of each thereof.
Dependencies are inferred from the claim text.