AAV-NAGLU vectors for treatment of mucopolysaccharidosis IIIB
AssigneeThe University of North Carolina at Chapel Hill
InventorsHaiyan Fu, Tierra Bobo
Life of this patent
Estimating the term…
Abstract
Recombinant nucleic acid comprising nucleotides encoding α-N-acetylglucosaminidase (NAGLU). Vectors comprising the recombinant nucleic acid for delivery to a subject. In an aspect the NAGLU sequences optimized for expression in human cells. Methods of using the vector to increase secretion of NAGLU from a cell and for treatment and prevention of mucopolysaccharidosis IIIB.
Claims19
A recombinant nucleic acid comprising a nucleotide sequence encoding human α-N-acetylglucosaminidase (NAGLU) that is codon-optimized for expression in human cells, wherein the recombinant nucleic acid comprises the nucleotide sequence of SEQ ID NO:
- 1.
Dependencies are inferred from the claim text.