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US 2019/0169618 A1Published application
Nucleic acids and methods for the treatment of pompe disease
AssigneeSynthena AG
InventorsLuis GARCIA, Aurelie AVRIL
Life of this patent
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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.
Claims20
A nucleic acid molecule 10 to 50 nucleotides in length, complementary to a nucleotide sequence of the acid alpha-glucosidase pre-mRNA, said nucleic acid being able to correct the splicing of exon 2 of the acid alpha-glucosidase pre-mRNA.
Dependencies are inferred from the claim text.