CA2857664A1 - Antisense oligonucleotides targeting within the smn2 pre-mrna for use ininduced exon inclusion in spinal muscle atrophy - Google Patents
Antisense oligonucleotides targeting within the smn2 pre-mrna for use ininduced exon inclusion in spinal muscle atrophy Download PDFInfo
- Publication number
- CA2857664A1 CA2857664A1 CA2857664A CA2857664A CA2857664A1 CA 2857664 A1 CA2857664 A1 CA 2857664A1 CA 2857664 A CA2857664 A CA 2857664A CA 2857664 A CA2857664 A CA 2857664A CA 2857664 A1 CA2857664 A1 CA 2857664A1
- Authority
- CA
- Canada
- Prior art keywords
- exon
- antisense oligonucleotide
- smn2
- group
- seq
- Prior art date
- Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
- Abandoned
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- 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
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- C12N15/09—Recombinant DNA-technology
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/111—General methods applicable to biologically active non-coding nucleic acids
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C—CHEMISTRY; METALLURGY
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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| US201161565499P | 2011-11-30 | 2011-11-30 | |
| US61/565,499 | 2011-11-30 | ||
| PCT/US2012/067475 WO2013082551A1 (en) | 2011-11-30 | 2012-11-30 | Induced exon inclusion in spinal muscle atrophy |
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| CA2857664A1 true CA2857664A1 (en) | 2013-06-06 |
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| CA2857664A Abandoned CA2857664A1 (en) | 2011-11-30 | 2012-11-30 | Antisense oligonucleotides targeting within the smn2 pre-mrna for use ininduced exon inclusion in spinal muscle atrophy |
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| US8802642B2 (en) | 2010-04-28 | 2014-08-12 | Iowa State University Research Foundation, Inc. | Spinal muscular atrophy treatment via targeting SMN2 catalytic core |
| JP6317675B2 (ja) | 2011-11-30 | 2018-04-25 | サレプタ セラピューティクス， インコーポレイテッド | 延長リピート病を処置するためのオリゴヌクレオチド |
| ES2727481T3 (es) | 2011-11-30 | 2019-10-16 | Sarepta Therapeutics Inc | Inclusión inducida de exón en atrofia muscular espinal |
| EP2946013A1 (en) | 2013-01-16 | 2015-11-25 | Iowa State University Research Foundation, Inc. | A deep intronic target for splicing correction on spinal muscular atrophy gene |
| JP6618910B2 (ja) * | 2013-09-05 | 2019-12-11 | サレプタ セラピューティクス，インコーポレイテッド | 酸性α−グルコシダーゼにおけるアンチセンス誘導エクソン２包含 |
| BR112017004056A2 (pt) | 2014-09-12 | 2017-12-05 | Biogen Ma Inc | composições e métodos para detecção da proteína smn em um indivíduo e tratamento de um indivíduo |
| US10905709B2 (en) * | 2015-08-28 | 2021-02-02 | Sarepta Therapeutics, Inc. | Modified antisense oligomers for exon inclusion in spinal muscular atrophy |
| US10357543B2 (en) | 2015-11-16 | 2019-07-23 | Ohio State Innovation Foundation | Methods and compositions for treating disorders and diseases using Survival Motor Neuron (SMN) protein |
| SG11201808964PA (en) | 2016-04-18 | 2018-11-29 | Sarepta Therapeutics Inc | Antisense oligomers and methods of using the same for treating diseases associated with the acid alpha-glucosidase gene |
| MX384727B (es) * | 2016-05-24 | 2025-03-14 | Sarepta Therapeutics Inc | Procesos para preparar oligómeros |
| MA45362A (fr) * | 2016-05-24 | 2019-04-10 | Sarepta Therapeutics Inc | Procédés de préparation d'oligomères morpholino de phosphorodiamidate |
| US11472824B2 (en) | 2016-05-24 | 2022-10-18 | Sarepta Therapeutics, Inc. | Processes for preparing phosphorodiamidate morpholino oligomers |
| IL263044B2 (en) | 2016-05-24 | 2024-06-01 | Sarepta Therapeutics Inc | Processes for preparing phosphorodiamidate morpholino oligomers |
| CA3024178A1 (en) | 2016-05-24 | 2017-12-14 | Sarepta Therapeutics, Inc. | Pharmaceutical composition comprising eteplirsen |
| JP6987081B2 (ja) | 2016-05-24 | 2021-12-22 | サレプタ セラピューティクス， インコーポレイテッド | ホスホロジアミダートモルホリノオリゴマーを調製するためのプロセス |
| WO2017218884A1 (en) | 2016-06-16 | 2017-12-21 | Ionis Pharmaceuticals, Inc. | Combinations for the modulation of smn expression |
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| EP2785840B1 (en) | 2019-03-20 |
| JP2015504650A (ja) | 2015-02-16 |
| JP2019059769A (ja) | 2019-04-18 |
| US10577605B2 (en) | 2020-03-03 |
| AU2012345638B2 (en) | 2018-07-12 |
| ES2727481T3 (es) | 2019-10-16 |
| US20140329772A1 (en) | 2014-11-06 |
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