Therapeutic adeno-associated virus for treating pompe disease

The recombinant AAV vector of claim 34, wherein the promoter is constitutive, cell specific or inducible.

-31. (canceled)

The recombinant AAV vector of claim 34, wherein the AAV3b serotype comprises one or mutations in a capsid protein selected from any of:

• 265D, 549A, Q263Y

The recombinant AAV vector of claim 34, wherein the AAV3b serotype is selected from any of:

• AAV3b265D, AAV3b265D549A, AAV3b549A or AAV3bQ263Y, or AAV3bSASTG.

A recombinant adenovirus associated (AAV) vector comprising in its genome:

• a. 5′ and 3′ AAV inverted terminal repeats (ITR) sequences, and
• b. located between the 5′ and 3′ ITRs, a heterologous nucleic acid sequence encoding a polypeptide comprising an alpha-glucosidase (GAA) polypeptide, wherein the heterologous nucleic acid is operatively linked to a liver specific promoter.
• wherein the recombinant AAV vector comprises a capsid protein of the AAV3b or AAV8 serotype.

(canceled)

-48. (canceled)

(canceled)

(canceled)

A nucleic acid sequence for a recombinant adenovirus associated viral (rAAV) vector genome comprising:

• a. 5′ and 3′ AAV inverted terminal repeats (ITR) nucleic acid sequences, and
• b. located between the 5′ and 3′ ITR sequence, a heterologous nucleic acid sequence encoding a fusion polypeptide comprising a secretory signal peptide and an alpha-glucosidase (GAA) polypeptide, wherein the heterologous nucleic acid is operatively linked to a promoter.

-68. (canceled)

-71. (canceled)