Back to results
US 2021/0277362 A1Published application
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.
Claims36
A recombinant adeno-associated virus (rAAV) comprises a polynucleotide which comprises a first AAV inverted terminal repeat (ITR), a promoter, a nucleotide sequence encoding a protein with calpain 3 (CAPN3) activity and a second AAV ITR.
Dependencies are inferred from the claim text.