Antisense oligonucleotides useful in treatment of Pompe disease
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..
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Patent| Erscheinungsjahr: |
2021 |
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| Erschienen: |
2021 |
| Enthalten in: |
Europäisches Patentamt - (2021) vom: 28. Okt. Zur Gesamtaufnahme - year:2021 |
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| Sprache: |
Englisch |
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| Beteiligte Personen: |
BERGSMA ATZE JACOBUS [VerfasserIn] |
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| Links: |
Volltext [kostenfrei] |
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| Anmerkungen: |
Source: www.epo.org (no modifications made), First posted: 2021-10-28, Last update posted on www.tib.eu: 2024-01-22, Last updated: 2024-01-26 |
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| Patentnummer: |
AU2015272128 |
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| Förderinstitution / Projekttitel: |
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| PPN (Katalog-ID): |
EPA005451221 |
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| 520 | |a 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. | ||
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