BR112020016524A2 - Terapia com oligonucleotídeos para a doença de wilson - Google Patents
Terapia com oligonucleotídeos para a doença de wilson Download PDFInfo
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- BR112020016524A2 BR112020016524A2 BR112020016524-7A BR112020016524A BR112020016524A2 BR 112020016524 A2 BR112020016524 A2 BR 112020016524A2 BR 112020016524 A BR112020016524 A BR 112020016524A BR 112020016524 A2 BR112020016524 A2 BR 112020016524A2
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- Prior art keywords
- antisense oligonucleotide
- atp7b
- seq
- exon
- sequence
- Prior art date
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Classifications
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- C—CHEMISTRY; METALLURGY
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- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
- C12N15/1137—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing against enzymes
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- A—HUMAN NECESSITIES
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- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K31/00—Medicinal preparations containing organic active ingredients
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- A61K31/7088—Compounds having three or more nucleosides or nucleotides
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- A—HUMAN NECESSITIES
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- C12N2310/33—Chemical structure of the base
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/35—Nature of the modification
- C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
- C12N2310/3525—MOE, methoxyethoxy
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- C12Y306/00—Hydrolases acting on acid anhydrides (3.6)
- C12Y306/03—Hydrolases acting on acid anhydrides (3.6) acting on acid anhydrides; catalysing transmembrane movement of substances (3.6.3)
- C12Y306/03004—Cu2+-exporting ATPase (3.6.3.4)
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| PCT/US2019/018076 WO2019161105A1 (en) | 2018-02-14 | 2019-02-14 | Oligonucleotide therapy for wilson disease |
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| CN117925624B (zh) * | 2024-03-22 | 2024-05-31 | 上海凌医生物科技有限公司 | 一种金属响应调控元件 |
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| EP3752614A1 (en) | 2020-12-23 |
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