CODON-OPTIMIZED ACID ALPHA-GLUCOSIDASE EXPRESSION CASSETTES AND METHODS OF USING SAME

CODON-OPTIMIZED ACID ALPHA-GLUCOSIDASE EXPRESSION CASSETTES AND METHODS OF USING SAME

The invention provides nucleic acids encoding acid α-glucosidase (GAA). In certain embodiments, nucleic acids have greater than about 86% sequence identity to a sequence selected from the group consisting of any of the sequences set forth as SEQ ID NOs:1-5. In certain embodiments, nucleic acids enco...

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Bibliographische Detailangaben
Hauptverfasser: ARMOUR, Sean, NORDIN, Jayme, ANGUELA, Xavier
Format: Patent
Sprache:eng ; fre ; ger
Schlagworte:
CHEMISTRY
HUMAN NECESSITIES
HYGIENE
MEDICAL OR VETERINARY SCIENCE
METALLURGY
ORGANIC CHEMISTRY
PEPTIDES
PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
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Beschreibung
Zusammenfassung:The invention provides nucleic acids encoding acid α-glucosidase (GAA). In certain embodiments, nucleic acids have greater than about 86% sequence identity to a sequence selected from the group consisting of any of the sequences set forth as SEQ ID NOs:1-5. In certain embodiments, nucleic acids encoding acid α-glucosidase (GAA) contain less than 127 CpG dinucleotides. Expression cassettes, vectors, cells and cell lines and methods of using such nucleic acids encoding acid α-glucosidase (GAA) are also provided.