JP2012530715A5 - - Google Patents
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- JP2012530715A5 JP2012530715A5 JP2012516315A JP2012516315A JP2012530715A5 JP 2012530715 A5 JP2012530715 A5 JP 2012530715A5 JP 2012516315 A JP2012516315 A JP 2012516315A JP 2012516315 A JP2012516315 A JP 2012516315A JP 2012530715 A5 JP2012530715 A5 JP 2012530715A5
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- Prior art keywords
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- nucleobase sequence
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- 239000008194 pharmaceutical composition Substances 0.000 claims 28
- 101000617738 Homo sapiens Survival motor neuron protein Proteins 0.000 claims 9
- 102100021947 Survival motor neuron protein Human genes 0.000 claims 9
- 108091034117 Oligonucleotide Proteins 0.000 claims 8
- 239000000074 antisense oligonucleotide Substances 0.000 claims 6
- 238000012230 antisense oligonucleotides Methods 0.000 claims 6
- 230000000692 anti-sense effect Effects 0.000 claims 5
- 150000001875 compounds Chemical class 0.000 claims 5
- 101100203485 Homo sapiens SMN2 gene Proteins 0.000 claims 3
- 230000000295 complement effect Effects 0.000 claims 3
- 102000053564 human SMN2 Human genes 0.000 claims 3
- 108020004999 messenger RNA Proteins 0.000 claims 3
- 210000002161 motor neuron Anatomy 0.000 claims 3
- 108020004707 nucleic acids Proteins 0.000 claims 3
- 102000039446 nucleic acids Human genes 0.000 claims 3
- 150000007523 nucleic acids Chemical class 0.000 claims 3
- 239000002777 nucleoside Substances 0.000 claims 3
- 150000003833 nucleoside derivatives Chemical class 0.000 claims 3
- 238000007910 systemic administration Methods 0.000 claims 3
- 210000003169 central nervous system Anatomy 0.000 claims 2
- 210000001175 cerebrospinal fluid Anatomy 0.000 claims 2
- 238000001802 infusion Methods 0.000 claims 2
- 208000024891 symptom Diseases 0.000 claims 2
- RYYWUUFWQRZTIU-UHFFFAOYSA-K thiophosphate Chemical compound [O-]P([O-])([O-])=S RYYWUUFWQRZTIU-UHFFFAOYSA-K 0.000 claims 2
- LRSASMSXMSNRBT-UHFFFAOYSA-N 5-methylcytosine Chemical group CC1=CNC(=O)N=C1N LRSASMSXMSNRBT-UHFFFAOYSA-N 0.000 claims 1
- 150000001413 amino acids Chemical class 0.000 claims 1
- 230000037396 body weight Effects 0.000 claims 1
- 210000004556 brain Anatomy 0.000 claims 1
- 201000010099 disease Diseases 0.000 claims 1
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims 1
- 239000007928 intraperitoneal injection Substances 0.000 claims 1
- 238000010253 intravenous injection Methods 0.000 claims 1
- 238000000034 method Methods 0.000 claims 1
- 229920001184 polypeptide Polymers 0.000 claims 1
- 102000004196 processed proteins & peptides Human genes 0.000 claims 1
- 108090000765 processed proteins & peptides Proteins 0.000 claims 1
- 210000002330 subarachnoid space Anatomy 0.000 claims 1
- 230000004083 survival effect Effects 0.000 claims 1
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US21803109P | 2009-06-17 | 2009-06-17 | |
| US61/218,031 | 2009-06-17 | ||
| PCT/US2010/039077 WO2010148249A1 (en) | 2009-06-17 | 2010-06-17 | Compositions and methods for modulation of smn2 splicing in a subject |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2015040152A Division JP6064139B2 (ja) | 2009-06-17 | 2015-03-02 | 被験体におけるｓｍｎ２スプライシングのモジュレーションのための組成物および方法 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2012530715A JP2012530715A (ja) | 2012-12-06 |
| JP2012530715A5 true JP2012530715A5 (enExample) | 2013-08-01 |
| JP5707396B2 JP5707396B2 (ja) | 2015-04-30 |
Family
ID=43356760
Family Applications (7)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2012516315A Active JP5707396B2 (ja) | 2009-06-17 | 2010-06-17 | 被験体におけるｓｍｎ２スプライシングのモジュレーションのための組成物および方法 |
| JP2015040152A Active JP6064139B2 (ja) | 2009-06-17 | 2015-03-02 | 被験体におけるｓｍｎ２スプライシングのモジュレーションのための組成物および方法 |
| JP2016216200A Active JP6370860B2 (ja) | 2009-06-17 | 2016-11-04 | 被験体におけるｓｍｎ２スプライシングのモジュレーションのための組成物および方法 |
| JP2018128926A Active JP6637121B2 (ja) | 2009-06-17 | 2018-07-06 | 被験体におけるｓｍｎ２スプライシングのモジュレーションのための組成物および方法 |
| JP2019227997A Pending JP2020073498A (ja) | 2009-06-17 | 2019-12-18 | 被験体におけるｓｍｎ２スプライシングのモジュレーションのための組成物および方法 |
| JP2021180965A Pending JP2022025131A (ja) | 2009-06-17 | 2021-11-05 | 被験体におけるｓｍｎ２スプライシングのモジュレーションのための組成物および方法 |
| JP2023185174A Pending JP2024012416A (ja) | 2009-06-17 | 2023-10-30 | 被験体におけるｓｍｎ２スプライシングのモジュレーションのための組成物および方法 |
Family Applications After (6)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2015040152A Active JP6064139B2 (ja) | 2009-06-17 | 2015-03-02 | 被験体におけるｓｍｎ２スプライシングのモジュレーションのための組成物および方法 |
| JP2016216200A Active JP6370860B2 (ja) | 2009-06-17 | 2016-11-04 | 被験体におけるｓｍｎ２スプライシングのモジュレーションのための組成物および方法 |
| JP2018128926A Active JP6637121B2 (ja) | 2009-06-17 | 2018-07-06 | 被験体におけるｓｍｎ２スプライシングのモジュレーションのための組成物および方法 |
| JP2019227997A Pending JP2020073498A (ja) | 2009-06-17 | 2019-12-18 | 被験体におけるｓｍｎ２スプライシングのモジュレーションのための組成物および方法 |
| JP2021180965A Pending JP2022025131A (ja) | 2009-06-17 | 2021-11-05 | 被験体におけるｓｍｎ２スプライシングのモジュレーションのための組成物および方法 |
| JP2023185174A Pending JP2024012416A (ja) | 2009-06-17 | 2023-10-30 | 被験体におけるｓｍｎ２スプライシングのモジュレーションのための組成物および方法 |
Country Status (25)
| Country | Link |
|---|---|
| US (5) | US8980853B2 (enExample) |
| EP (4) | EP3643783A1 (enExample) |
| JP (7) | JP5707396B2 (enExample) |
| KR (8) | KR20170054537A (enExample) |
| CN (5) | CN115227710A (enExample) |
| AU (2) | AU2010262862C1 (enExample) |
| CA (1) | CA2765396C (enExample) |
| CY (2) | CY1120807T1 (enExample) |
| DK (2) | DK3305302T3 (enExample) |
| ES (2) | ES2761614T3 (enExample) |
| HK (1) | HK1246648B (enExample) |
| HR (2) | HRP20181712T1 (enExample) |
| HU (2) | HUE046966T2 (enExample) |
| IL (2) | IL217014A (enExample) |
| LT (2) | LT3305302T (enExample) |
| MX (3) | MX361732B (enExample) |
| NO (1) | NO2020008I1 (enExample) |
| NZ (2) | NZ597071A (enExample) |
| PL (2) | PL3449926T3 (enExample) |
| PT (2) | PT3449926T (enExample) |
| RU (2) | RU2566724C9 (enExample) |
| SI (2) | SI3449926T1 (enExample) |
| SM (2) | SMT202000020T1 (enExample) |
| TR (1) | TR201816256T4 (enExample) |
| WO (1) | WO2010148249A1 (enExample) |
Families Citing this family (116)
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