THERAPEUTIC ADENO-ASSOCIATED VIRUS COMPRISING LIVER-SPECIFIC PROMOTERS FOR TREATING POMPE DISEASE AND LYSOSOMAL DISORDERS

Recombinant AAV (rAAV) vectors comprising a rAVV genome comprising a heterologous nucleic acid encoding a lysosomal protein, e.g., acid alpha-glucosidase (GAA) polypeptide, and optionally a signal peptide and/or optionally a targeting sequence, e.g., IGF2 targeting peptide, operatively linked to a l...

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Hauptverfasser:	IGLESIAS, Juan Manuel, FRANCOIS, Achille, TRETIAKOVA, Anna, O'CALLAGHAN, Michael W, ROBERTS, Michael
Format:	Patent
Sprache:	eng ; fre ; ger
Schlagworte:	BEER BIOCHEMISTRY CHEMISTRY COMPOSITIONS THEREOF CULTURE MEDIA ENZYMOLOGY METALLURGY MICROBIOLOGY MICROORGANISMS OR ENZYMES MUTATION OR GENETIC ENGINEERING PROPAGATING, PRESERVING OR MAINTAINING MICROORGANISMS SPIRITS VINEGAR WINE
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Beschreibung
Zusammenfassung:	Recombinant AAV (rAAV) vectors comprising a rAVV genome comprising a heterologous nucleic acid encoding a lysosomal protein, e.g., acid alpha-glucosidase (GAA) polypeptide, and optionally a signal peptide and/or optionally a targeting sequence, e.g., IGF2 targeting peptide, operatively linked to a liver-specific promoter (LSP), enabling the GAA polypeptide to be secreted from the liver and targeted to the lysosomes. Particular embodiments relate to a recombinant AAV (rAAV) vector encoding an alpha-glucosidase (GAA) polypeptide, having a liver secretory signal peptide and a IGF2 targeting peptide that binds human cation-independent mannose-6-phosphate receptor (CI-MPR) or to the IGF2 receptor, permitting proper subcellular localization of the GAA polypeptide to lysosomes. Also encompassed are cells, and methods to treat a lysosomal disease, for example, a glycogen storage disease type II (GSD II) disease and/or Pompe Disease with the rAAV vector.