Antisense oligonucleotides useful in treatment of pompe disease
AssigneeErasmus University Medical Center Rotterdam
InventorsAtze Jacobus BERGSMA, Erik van der Wal, Wilhelmus Wenceslaus Matthias Pijnappel, Antje Tjitske van der Ploeg, Arnoldus REUSER
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Abstract
The present invention is directed to antisense oligomeric compounds that may be used in the treatment Pompe disease as well as method for modulating the splicing of the GAA gene and method to treat Pompe disease. Also pharmaceutical compositions comprising the antisense oligomeric compounds are part of the invention.
Claims22
An antisense oligomeric compound targeting SEQ ID NO:
- 1 and single nucleotide polymorphism of SEQ ID NO:
- 1, wherein at least one of the nucleotides is modified.
(canceled)
(canceled)
A method of modulating splicing of GAA pre-mRNA in a cell comprising:
- contacting the cell with an antisense oligomeric compound targeting SEQ ID NO:1 and single nucleotide polymorphism of SEQ ID NO:1.
A method for treating Pompe disease in a patient comprising administering an effective amount of an antisense oligomeric compound targeting SEQ ID NO:1 and single nucleotide polymorphism of SEQ ID NO:1.
A method to restore the function of GAA in a cell wherein said method comprises the administration of an antisense oligomeric compound targeting SEQ ID NO:1 and single nucleotide polymorphism of SEQ ID NO:1.
A method of correcting abnormal gene expression in a cell, preferably a muscular cell, of a subject, the method comprising administering to the subject an antisense oligomeric compound targeting SEQ ID NO:1 and single nucleotide polymorphism of SEQ ID NO:1.
A compound capable of binding to a genomic nucleic acid sequence of GAA gene targeting SEQ ID NO:
- 1.
Dependencies are inferred from the claim text.