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publicationDate 2016-07-28-04:00^^<http://www.w3.org/2001/XMLSchema#date>
publicationNumber US-2016215291-A1
titleOfInvention Nucleic acids and methods for the treatment of pompe disease
abstract The invention relates to nucleic acids and methods for restoring acid alpha-glucosidase (GAA) activity in patients with Pompe disease using splice-switching technology.
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