Patent ID: 11920149
Assignee: VRIJE UNIVERSITEIT BRUSSEL
Field: Pharmaceuticals (Chemistry)
Classification: CPC C  A | IPC A  C

Claim 16:
17. A method of treating Pompe disease comprising administering a therapeutically effective amount of the pharmaceutical composition according to claim 15 to a subject in need thereof, wherein the vector comprises a CRE-CSk-SH5 regulatory element (SEQ ID NO: 122), a nucleic acid regulatory element having a maximal length of 1000 nucleotides and comprising the sequence defined by SEQ ID NO: 4, the complement of said sequence, or a sequence hybridizing under stringent conditions to said nucleic acid regulatory element, an SPc5-12 promoter, and a human GAA transgene (SEQ ID NO: 93) or a codon-optimised variant thereof (SEQ ID NO: 94).