JP2016533761A5 - - Google Patents
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- JP2016533761A5 JP2016533761A5 JP2016540437A JP2016540437A JP2016533761A5 JP 2016533761 A5 JP2016533761 A5 JP 2016533761A5 JP 2016540437 A JP2016540437 A JP 2016540437A JP 2016540437 A JP2016540437 A JP 2016540437A JP 2016533761 A5 JP2016533761 A5 JP 2016533761A5
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- 229910052739 hydrogen Inorganic materials 0.000 claims description 90
- 239000001257 hydrogen Substances 0.000 claims description 90
- 150000002431 hydrogen Chemical class 0.000 claims description 66
- 125000003710 aryl alkyl group Chemical group 0.000 claims description 60
- 125000000217 alkyl group Chemical group 0.000 claims description 57
- 230000008685 targeting Effects 0.000 claims description 43
- ISAKRJDGNUQOIC-UHFFFAOYSA-N Uracil Chemical group O=C1C=CNC(=O)N1 ISAKRJDGNUQOIC-UHFFFAOYSA-N 0.000 claims description 40
- RWQNBRDOKXIBIV-UHFFFAOYSA-N thymine Chemical compound CC1=CNC(=O)NC1=O RWQNBRDOKXIBIV-UHFFFAOYSA-N 0.000 claims description 40
- 150000001875 compounds Chemical class 0.000 claims description 33
- 102000020313 Cell-Penetrating Peptides Human genes 0.000 claims description 27
- 108010051109 Cell-Penetrating Peptides Proteins 0.000 claims description 27
- 125000003729 nucleotide group Chemical group 0.000 claims description 26
- UFHFLCQGNIYNRP-UHFFFAOYSA-N Hydrogen Chemical compound [H][H] UFHFLCQGNIYNRP-UHFFFAOYSA-N 0.000 claims description 24
- 239000002773 nucleotide Substances 0.000 claims description 20
- 229940113082 thymine Drugs 0.000 claims description 20
- 229940035893 uracil Drugs 0.000 claims description 20
- 125000002252 acyl group Chemical group 0.000 claims description 18
- 125000002496 methyl group Chemical group [H]C([H])([H])* 0.000 claims description 18
- 230000000692 anti-sense effect Effects 0.000 claims description 16
- FWMNVWWHGCHHJJ-SKKKGAJSSA-N 4-amino-1-[(2r)-6-amino-2-[[(2r)-2-[[(2r)-2-[[(2r)-2-amino-3-phenylpropanoyl]amino]-3-phenylpropanoyl]amino]-4-methylpentanoyl]amino]hexanoyl]piperidine-4-carboxylic acid Chemical compound C([C@H](C(=O)N[C@H](CC(C)C)C(=O)N[C@H](CCCCN)C(=O)N1CCC(N)(CC1)C(O)=O)NC(=O)[C@H](N)CC=1C=CC=CC=1)C1=CC=CC=C1 FWMNVWWHGCHHJJ-SKKKGAJSSA-N 0.000 claims description 14
- 101001018026 Homo sapiens Lysosomal alpha-glucosidase Proteins 0.000 claims description 14
- 102000045921 human GAA Human genes 0.000 claims description 14
- 108020004999 messenger RNA Proteins 0.000 claims description 14
- 108090000623 proteins and genes Proteins 0.000 claims description 14
- 239000008194 pharmaceutical composition Substances 0.000 claims description 13
- 108091093037 Peptide nucleic acid Proteins 0.000 claims description 12
- QVGXLLKOCUKJST-UHFFFAOYSA-N atomic oxygen Chemical group [O] QVGXLLKOCUKJST-UHFFFAOYSA-N 0.000 claims description 12
- 229910052736 halogen Inorganic materials 0.000 claims description 12
- 150000002367 halogens Chemical class 0.000 claims description 12
- 125000002887 hydroxy group Chemical group [H]O* 0.000 claims description 12
- 229910052757 nitrogen Inorganic materials 0.000 claims description 12
- 125000004433 nitrogen atom Chemical group N* 0.000 claims description 12
- 125000001997 phenyl group Chemical group [H]C1=C([H])C([H])=C(*)C([H])=C1[H] 0.000 claims description 12
- 125000001424 substituent group Chemical group 0.000 claims description 12
- 125000002221 trityl group Chemical group [H]C1=C([H])C([H])=C([H])C([H])=C1C([*])(C1=C(C(=C(C(=C1[H])[H])[H])[H])[H])C1=C([H])C([H])=C([H])C([H])=C1[H] 0.000 claims description 12
- 230000000295 complement effect Effects 0.000 claims description 10
- 208000007345 glycogen storage disease Diseases 0.000 claims description 8
- 150000003839 salts Chemical class 0.000 claims description 8
- 125000003545 alkoxy group Chemical group 0.000 claims description 6
- 125000003282 alkyl amino group Chemical group 0.000 claims description 6
- 125000004429 atom Chemical group 0.000 claims description 6
- 229910052799 carbon Inorganic materials 0.000 claims description 6
- 125000002915 carbonyl group Chemical group [*:2]C([*:1])=O 0.000 claims description 6
- 108020004707 nucleic acids Proteins 0.000 claims description 6
- 102000039446 nucleic acids Human genes 0.000 claims description 6
- 150000007523 nucleic acids Chemical class 0.000 claims description 6
- 229910052760 oxygen Inorganic materials 0.000 claims description 6
- 239000001301 oxygen Substances 0.000 claims description 6
- 239000000126 substance Substances 0.000 claims description 6
- RYYWUUFWQRZTIU-UHFFFAOYSA-K thiophosphate Chemical compound [O-]P([O-])([O-])=S RYYWUUFWQRZTIU-UHFFFAOYSA-K 0.000 claims description 6
- -1 C 1 -C 6 alkyl Substances 0.000 claims description 5
- OPTASPLRGRRNAP-UHFFFAOYSA-N cytosine Chemical compound NC=1C=CNC(=O)N=1 OPTASPLRGRRNAP-UHFFFAOYSA-N 0.000 claims description 4
- 239000003937 drug carrier Substances 0.000 claims description 4
- UYTPUPDQBNUYGX-UHFFFAOYSA-N guanine Chemical compound O=C1NC(N)=NC2=C1N=CN2 UYTPUPDQBNUYGX-UHFFFAOYSA-N 0.000 claims description 4
- FDGQSTZJBFJUBT-UHFFFAOYSA-N hypoxanthine Chemical compound O=C1NC=NC2=C1NC=N2 FDGQSTZJBFJUBT-UHFFFAOYSA-N 0.000 claims description 4
- IZGFERLHOCMZQF-UHFFFAOYSA-N 2-(10h-phenoxazin-1-yloxy)ethanamine Chemical compound O1C2=CC=CC=C2NC2=C1C=CC=C2OCCN IZGFERLHOCMZQF-UHFFFAOYSA-N 0.000 claims description 2
- LRSASMSXMSNRBT-UHFFFAOYSA-N 5-methylcytosine Chemical compound CC1=CNC(=O)N=C1N LRSASMSXMSNRBT-UHFFFAOYSA-N 0.000 claims description 2
- MSSXOMSJDRHRMC-UHFFFAOYSA-N 9H-purine-2,6-diamine Chemical compound NC1=NC(N)=C2NC=NC2=N1 MSSXOMSJDRHRMC-UHFFFAOYSA-N 0.000 claims description 2
- GFFGJBXGBJISGV-UHFFFAOYSA-N Adenine Chemical compound NC1=NC=NC2=C1N=CN2 GFFGJBXGBJISGV-UHFFFAOYSA-N 0.000 claims description 2
- 229930024421 Adenine Natural products 0.000 claims description 2
- UGQMRVRMYYASKQ-UHFFFAOYSA-N Hypoxanthine nucleoside Natural products OC1C(O)C(CO)OC1N1C(NC=NC2=O)=C2N=C1 UGQMRVRMYYASKQ-UHFFFAOYSA-N 0.000 claims description 2
- 229960000643 adenine Drugs 0.000 claims description 2
- 229940104302 cytosine Drugs 0.000 claims description 2
- 239000000203 mixture Substances 0.000 claims 7
- 108090000765 processed proteins & peptides Proteins 0.000 claims 3
- 238000000034 method Methods 0.000 description 6
- 0 CN1C*CCCC1 Chemical compound CN1C*CCCC1 0.000 description 2
- LJLWVVCWBURGCC-UHFFFAOYSA-N CC(C)C(C)NC Chemical compound CC(C)C(C)NC LJLWVVCWBURGCC-UHFFFAOYSA-N 0.000 description 1
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201361874261P | 2013-09-05 | 2013-09-05 | |
| US61/874,261 | 2013-09-05 | ||
| US201461932195P | 2014-01-27 | 2014-01-27 | |
| US61/932,195 | 2014-01-27 | ||
| PCT/US2014/054384 WO2015035231A1 (en) | 2013-09-05 | 2014-09-05 | Antisense-induced exon2 inclusion in acid alpha-glucosidase |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2018227359A Division JP6671449B2 (ja) | 2013-09-05 | 2018-12-04 | 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 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2016533761A JP2016533761A (ja) | 2016-11-04 |
| JP2016533761A5 true JP2016533761A5 (enExample) | 2017-10-12 |
| JP6618910B2 JP6618910B2 (ja) | 2019-12-11 |
Family
ID=51541398
Family Applications (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2016540437A Active JP6618910B2 (ja) | 2013-09-05 | 2014-09-05 | 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 |
| JP2018227359A Active JP6671449B2 (ja) | 2013-09-05 | 2018-12-04 | 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 |
| JP2020035519A Active JP6865871B2 (ja) | 2013-09-05 | 2020-03-03 | 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 |
Family Applications After (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2018227359A Active JP6671449B2 (ja) | 2013-09-05 | 2018-12-04 | 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 |
| JP2020035519A Active JP6865871B2 (ja) | 2013-09-05 | 2020-03-03 | 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 |
Country Status (11)
| Country | Link |
|---|---|
| US (3) | US11236338B2 (enExample) |
| EP (1) | EP3041935A1 (enExample) |
| JP (3) | JP6618910B2 (enExample) |
| KR (2) | KR20210088009A (enExample) |
| CN (2) | CN111235149B (enExample) |
| AU (2) | AU2014317961B2 (enExample) |
| CA (1) | CA2922838A1 (enExample) |
| IL (4) | IL300444A (enExample) |
| MX (1) | MX384377B (enExample) |
| TW (1) | TWI736514B (enExample) |
| WO (1) | WO2015035231A1 (enExample) |
Families Citing this family (26)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2006086667A2 (en) | 2005-02-09 | 2006-08-17 | Avi Bio Pharma, Inc. | Antisense composition and method for treating muscle atrophy |
| US20130085139A1 (en) | 2011-10-04 | 2013-04-04 | Royal Holloway And Bedford New College | Oligomers |
| AU2014317961B2 (en) | 2013-09-05 | 2020-07-30 | Murdoch University | Antisense-induced exon2 inclusion in acid alpha-glucosidase |
| DK3044315T3 (da) * | 2013-09-11 | 2019-05-20 | Synthena Ag | Nukleinsyrer og fremgangsmåder til behandling af pompes sygdom |
| EP4039807A1 (en) * | 2014-06-10 | 2022-08-10 | Erasmus University Rotterdam Medical Center | Antisense oligonucleotides useful in treatment of pompe disease |
| GB201410693D0 (en) | 2014-06-16 | 2014-07-30 | Univ Southampton | Splicing modulation |
| CN107109411B (zh) | 2014-10-03 | 2022-07-01 | 冷泉港实验室 | 核基因输出的定向增加 |
| MX2017011004A (es) * | 2015-02-27 | 2018-02-09 | Sarepta Therapeutics Inc | Inclusion del exon2 inducida por antisentido en alfa-glucosidasa.acida. |
| MA41795A (fr) * | 2015-03-18 | 2018-01-23 | Sarepta Therapeutics Inc | Exclusion d'un exon induite par des composés antisens dans la myostatine |
| JP2018530560A (ja) * | 2015-10-09 | 2018-10-18 | サレプタ セラピューティクス， インコーポレイテッド | デュシェンヌ型筋ジストロフィーおよび関連障害の処置のための組成物および方法 |
| CN108603230A (zh) | 2015-10-09 | 2018-09-28 | 南安普敦大学 | 基因表达的调节与蛋白质表达失调的筛选 |
| US10993995B2 (en) | 2015-12-07 | 2021-05-04 | Erasmus University Medical Center Rotterdam | Enzymatic replacement therapy and antisense therapy for pompe disease |
| US11096956B2 (en) | 2015-12-14 | 2021-08-24 | Stoke Therapeutics, Inc. | Antisense oligomers and uses thereof |
| CN116059236A (zh) | 2015-12-14 | 2023-05-05 | 冷泉港实验室 | 用于治疗常染色体显性精神发育迟滞5型和Dravet综合征的反义寡聚体 |
| US11060089B2 (en) * | 2016-04-18 | 2021-07-13 | Sarepta Therapeutics, Inc. | Antisense oligomers and methods of using the same for treating diseases associated with the acid alpha-glucosidase gene |
| NL2017294B1 (en) * | 2016-08-05 | 2018-02-14 | Univ Erasmus Med Ct Rotterdam | Natural cryptic exon removal by pairs of antisense oligonucleotides. |
| NL2017295B1 (en) * | 2016-08-05 | 2018-02-14 | Univ Erasmus Med Ct Rotterdam | Antisense oligomeric compound for Pompe disease |
| NZ755416A (en) * | 2016-12-19 | 2023-05-26 | Sarepta Therapeutics Inc | Exon skipping oligomer conjugates for muscular dystrophy |
| KR20260009385A (ko) | 2017-08-25 | 2026-01-19 | 스톡 테라퓨틱스, 인크. | 병태 및 질환 치료용 안티센스 올리고머 |
| NL2019517B1 (en) * | 2017-09-08 | 2019-03-19 | Univ Erasmus Med Ct Rotterdam | New therapy for Pompe disease |
| TWI812647B (zh) | 2017-09-25 | 2023-08-21 | 美商薩羅塔治療公司 | 經由速流合成以製備磷醯二胺嗎啉代寡聚物之製程 |
| JP2021523227A (ja) | 2018-05-04 | 2021-09-02 | ストーク セラピューティクス，インク． | コレステリルエステル蓄積症の処置のための方法及び組成物 |
| KR20210134003A (ko) | 2019-02-27 | 2021-11-08 | 스톡 테라퓨틱스, 인크. | 병태 및 질환의 치료를 위한 안티센스 올리고머 |
| US12338437B2 (en) | 2020-05-11 | 2025-06-24 | Stoke Therapeutics, Inc. | OPA1 antisense oligomers for treatment of conditions and diseases |
| KR102934598B1 (ko) * | 2020-06-05 | 2026-03-05 | 넥스올리고(주) | 신규한 몰포리노 올리고뉴클레오티드 유도체 |
| WO2026080471A2 (en) | 2024-10-07 | 2026-04-16 | Flagship Pioneering Innovations Vi, Llc | Compositions of trem conjugates and uses thereof |
Family Cites Families (94)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US4426330A (en) | 1981-07-20 | 1984-01-17 | Lipid Specialties, Inc. | Synthetic phospholipid compounds |
| US4534899A (en) | 1981-07-20 | 1985-08-13 | Lipid Specialties, Inc. | Synthetic phospholipid compounds |
| US5185444A (en) | 1985-03-15 | 1993-02-09 | Anti-Gene Deveopment Group | Uncharged morpolino-based polymers having phosphorous containing chiral intersubunit linkages |
| US5521063A (en) | 1985-03-15 | 1996-05-28 | Antivirals Inc. | Polynucleotide reagent containing chiral subunits and methods of use |
| US5166315A (en) | 1989-12-20 | 1992-11-24 | Anti-Gene Development Group | Sequence-specific binding polymers for duplex nucleic acids |
| US5034506A (en) | 1985-03-15 | 1991-07-23 | Anti-Gene Development Group | Uncharged morpholino-based polymers having achiral intersubunit linkages |
| US5506337A (en) | 1985-03-15 | 1996-04-09 | Antivirals Inc. | Morpholino-subunit combinatorial library and method |
| JP2528107B2 (ja) | 1985-03-15 | 1996-08-28 | サマ−トン，ジエ−ムス | ポリヌクレオチド測定試薬と方法 |
| US5217866A (en) | 1985-03-15 | 1993-06-08 | Anti-Gene Development Group | Polynucleotide assay reagent and method |
| US5354844A (en) | 1989-03-16 | 1994-10-11 | Boehringer Ingelheim International Gmbh | Protein-polycation conjugates |
| US5108921A (en) | 1989-04-03 | 1992-04-28 | Purdue Research Foundation | Method for enhanced transmembrane transport of exogenous molecules |
| US5227170A (en) | 1989-06-22 | 1993-07-13 | Vestar, Inc. | Encapsulation process |
| US5356633A (en) | 1989-10-20 | 1994-10-18 | Liposome Technology, Inc. | Method of treatment of inflamed tissues |
| US5013556A (en) | 1989-10-20 | 1991-05-07 | Liposome Technology, Inc. | Liposomes with enhanced circulation time |
| US5527528A (en) | 1989-10-20 | 1996-06-18 | Sequus Pharmaceuticals, Inc. | Solid-tumor treatment method |
| US5580575A (en) | 1989-12-22 | 1996-12-03 | Imarx Pharmaceutical Corp. | Therapeutic drug delivery systems |
| US5469854A (en) | 1989-12-22 | 1995-11-28 | Imarx Pharmaceutical Corp. | Methods of preparing gas-filled liposomes |
| US5264618A (en) | 1990-04-19 | 1993-11-23 | Vical, Inc. | Cationic lipids for intracellular delivery of biologically active molecules |
| JP3220180B2 (ja) | 1991-05-23 | 2001-10-22 | 三菱化学株式会社 | 薬剤含有タンパク質結合リポソーム |
| US5719262A (en) | 1993-11-22 | 1998-02-17 | Buchardt, Deceased; Ole | Peptide nucleic acids having amino acid side chains |
| US5539082A (en) | 1993-04-26 | 1996-07-23 | Nielsen; Peter E. | Peptide nucleic acids |
| US5714331A (en) | 1991-05-24 | 1998-02-03 | Buchardt, Deceased; Ole | Peptide nucleic acids having enhanced binding affinity, sequence specificity and solubility |
| AU659482B2 (en) | 1991-06-28 | 1995-05-18 | Massachusetts Institute Of Technology | Localized oligonucleotide therapy |
| NZ244306A (en) | 1991-09-30 | 1995-07-26 | Boehringer Ingelheim Int | Composition for introducing nucleic acid complexes into eucaryotic cells, complex containing nucleic acid and endosomolytic agent, peptide with endosomolytic domain and nucleic acid binding domain and preparation |
| US5521291A (en) | 1991-09-30 | 1996-05-28 | Boehringer Ingelheim International, Gmbh | Conjugates for introducing nucleic acid into higher eucaryotic cells |
| FR2692265B1 (fr) | 1992-05-25 | 1996-11-08 | Centre Nat Rech Scient | Composes biologiquement actifs de type phosphotriesters. |
| US5583020A (en) | 1992-11-24 | 1996-12-10 | Ribozyme Pharmaceuticals, Inc. | Permeability enhancers for negatively charged polynucleotides |
| JP3351476B2 (ja) | 1993-01-22 | 2002-11-25 | 三菱化学株式会社 | リン脂質誘導体及びそれを含有するリポソーム |
| US5395619A (en) | 1993-03-03 | 1995-03-07 | Liposome Technology, Inc. | Lipid-polymer conjugates and liposomes |
| US5462854A (en) | 1993-04-19 | 1995-10-31 | Beckman Instruments, Inc. | Inverse linkage oligonucleotides for chemical and enzymatic processes |
| FR2705099B1 (fr) | 1993-05-12 | 1995-08-04 | Centre Nat Rech Scient | Oligonucléotides phosphorothioates triesters et procédé de préparation. |
| US5534259A (en) | 1993-07-08 | 1996-07-09 | Liposome Technology, Inc. | Polymer compound and coated particle composition |
| US5543158A (en) | 1993-07-23 | 1996-08-06 | Massachusetts Institute Of Technology | Biodegradable injectable nanoparticles |
| US5417978A (en) | 1993-07-29 | 1995-05-23 | Board Of Regents, The University Of Texas System | Liposomal antisense methyl phosphonate oligonucleotides and methods for their preparation and use |
| US5595756A (en) | 1993-12-22 | 1997-01-21 | Inex Pharmaceuticals Corporation | Liposomal compositions for enhanced retention of bioactive agents |
| US5543152A (en) | 1994-06-20 | 1996-08-06 | Inex Pharmaceuticals Corporation | Sphingosomes for enhanced drug delivery |
| US5591721A (en) | 1994-10-25 | 1997-01-07 | Hybridon, Inc. | Method of down-regulating gene expression |
| US5512295A (en) | 1994-11-10 | 1996-04-30 | The Board Of Trustees Of The Leland Stanford Junior University | Synthetic liposomes for enhanced uptake and delivery |
| WO1997022371A1 (en) | 1995-12-18 | 1997-06-26 | Collagen Corporation | Crosslinked polymer compositions and methods for their use |
| US6245747B1 (en) | 1996-03-12 | 2001-06-12 | The Board Of Regents Of The University Of Nebraska | Targeted site specific antisense oligodeoxynucleotide delivery method |
| US7572582B2 (en) | 1997-09-12 | 2009-08-11 | Exiqon A/S | Oligonucleotide analogues |
| US6794499B2 (en) | 1997-09-12 | 2004-09-21 | Exiqon A/S | Oligonucleotide analogues |
| US7084125B2 (en) | 1999-03-18 | 2006-08-01 | Exiqon A/S | Xylo-LNA analogues |
| ATE356824T1 (de) | 1999-05-04 | 2007-04-15 | Santaris Pharma As | L-ribo-lna analoge |
| US6287860B1 (en) | 2000-01-20 | 2001-09-11 | Isis Pharmaceuticals, Inc. | Antisense inhibition of MEKK2 expression |
| AU5752601A (en) * | 2000-05-04 | 2001-11-12 | Avi Biopharma Inc | Splice-region antisense composition and method |
| KR100408844B1 (ko) * | 2000-07-29 | 2003-12-06 | 한국산업기술평가원 | 동물세포 발현벡터 |
| US20040049022A1 (en) * | 2001-04-24 | 2004-03-11 | Nyce Jonathan W. | Composition & methods for treatment and screening |
| AU2002305236A1 (en) * | 2001-04-24 | 2002-11-05 | Epigenesis Pharmaceuticals, Inc. | Composition, formulations and kits for treatment of respiratory and lung disease with anti-sense oligonucleotides and a bronchodilating agent |
| US7060809B2 (en) | 2001-09-04 | 2006-06-13 | Exiqon A/S | LNA compositions and uses thereof |
| US6965025B2 (en) | 2001-12-10 | 2005-11-15 | Isis Pharmaceuticals, Inc. | Antisense modulation of connective tissue growth factor expression |
| KR100464261B1 (ko) | 2002-01-24 | 2005-01-03 | 주식회사 파나진 | Ｐｎａ 올리고머를 합성하기 위한 신규한 단량체 및 그의제조방법 |
| KR20030084444A (ko) | 2002-04-26 | 2003-11-01 | 주식회사 파나진 | Ｐｎａ 올리고머를 합성하기 위한 신규한 단량체 및 그의제조방법 |
| US7569575B2 (en) | 2002-05-08 | 2009-08-04 | Santaris Pharma A/S | Synthesis of locked nucleic acid derivatives |
| US7250496B2 (en) * | 2002-11-14 | 2007-07-31 | Rosetta Genomics Ltd. | Bioinformatically detectable group of novel regulatory genes and uses thereof |
| EP2305813A3 (en) * | 2002-11-14 | 2012-03-28 | Dharmacon, Inc. | Fuctional and hyperfunctional sirna |
| US7211668B2 (en) | 2003-07-28 | 2007-05-01 | Panagene, Inc. | PNA monomer and precursor |
| US20070155685A1 (en) * | 2004-02-27 | 2007-07-05 | Karl-Hermann Schlingensiepen | Pharmaceutical composition |
| US7838657B2 (en) | 2004-12-03 | 2010-11-23 | University Of Massachusetts | Spinal muscular atrophy (SMA) treatment via targeting of SMN2 splice site inhibitory sequences |
| MX2007007184A (es) * | 2005-05-05 | 2008-01-14 | Antisense Pharma Gmbh | Uso de bajas dosis de oligonucleotidos antisentido para tgf-beta, vegf, interleucina-10, c-jun, c-fos o genes e2 de prostaglandina en el tratamiento de tumores. |
| DE202005008426U1 (de) | 2005-05-31 | 2005-09-15 | Ricon Sieb Und Foerdertechnik | Fördervorrichtung mit Fördertuch |
| PT2548560E (pt) | 2005-06-23 | 2015-09-22 | Isis Pharmaceuticals Inc | Composições e métodos de modulação da excisão-união de smn2 |
| EP2239328A3 (en) * | 2005-08-18 | 2011-01-05 | Alnylam Pharmaceuticals Inc. | Methods and compositions for treating neurological disease |
| LT2735568T (lt) | 2006-05-10 | 2017-11-27 | Sarepta Therapeutics, Inc. | Oligonukleotido analogai, turintys katijonines jungtis tarp subvienetų |
| US20100016215A1 (en) | 2007-06-29 | 2010-01-21 | Avi Biopharma, Inc. | Compound and method for treating myotonic dystrophy |
| JP2010533170A (ja) | 2007-07-12 | 2010-10-21 | プロセンサ テクノロジーズ ビー．ブイ． | 化合物を種々の選択された臓器、組織又は腫瘍細胞に標的化するための分子 |
| US7935816B2 (en) | 2007-10-25 | 2011-05-03 | Gene Tools, Llc | Molecular transporter compositions comprising dendrimeric oligoguanidine with a triazine core that facilitate delivery into cells in vivo |
| MX2010004955A (es) | 2007-11-15 | 2010-06-30 | Avi Biopharma Inc | Metodo de sintesis de oligomeros de morfolina. |
| SI2607484T1 (sl) * | 2008-10-27 | 2016-11-30 | Biomarin Technologies B.V. | Metode in sredstva za učinkovito preskakovanje eksona 45 v pre-mRNA Duchennove mišične distrofije |
| CA2758189C (en) | 2009-04-10 | 2020-12-29 | Association Institut De Myologie | Tricyclo-dna antisense oligonucleotides, compositions, and methods for the treatment of disease |
| US20120149757A1 (en) | 2009-04-13 | 2012-06-14 | Krainer Adrian R | Compositions and methods for modulation of smn2 splicing |
| KR20220084437A (ko) | 2009-06-17 | 2022-06-21 | 바이오젠 엠에이 인코포레이티드 | 대상에게서 ｓｍｎ２ 스플라이싱을 조정하기 위한 조성물 및 방법 |
| US8536148B2 (en) * | 2009-09-04 | 2013-09-17 | The United States Of America, As Represented By The Secretary, Department Of Health And Human Services | Disabling autophagy as a treatment for lysosomal storage diseases |
| US8921334B2 (en) * | 2009-12-29 | 2014-12-30 | Curna, Inc. | Treatment of nuclear respiratory factor 1 (NRF1) related diseases by inhibition of natural antisense transcript to NRF1 |
| FR2957090A1 (fr) * | 2010-03-05 | 2011-09-09 | Immunosearch | Methode d'evaluation du potentiel irritant d'un compose test |
| US8802642B2 (en) | 2010-04-28 | 2014-08-12 | Iowa State University Research Foundation, Inc. | Spinal muscular atrophy treatment via targeting SMN2 catalytic core |
| KR101981705B1 (ko) | 2010-05-28 | 2019-05-24 | 사렙타 쎄러퓨틱스, 인코퍼레이티드 | 변형된 서브유니트간 결합 및/또는 말단 그룹을 갖는 올리고뉴클레오타이드 유사체 |
| CN103118694B (zh) * | 2010-06-01 | 2016-08-03 | Atyr医药公司 | 与赖氨酰-tRNA合成酶的蛋白片段相关的治疗、诊断和抗体组合物的发现 |
| CN103154009B (zh) | 2010-09-30 | 2015-06-10 | 日本新药株式会社 | 吗啉基核酸衍生物 |
| WO2012092645A1 (en) * | 2011-01-04 | 2012-07-12 | Co-Operative Research Centre For Asthma And Airways | Novel antisense oligonucleotides |
| AU2011367230B2 (en) | 2011-05-05 | 2017-08-10 | Sarepta Therapeutics, Inc. | Peptide oligonucleotide conjugates |
| DK2581448T3 (en) | 2011-10-13 | 2015-04-27 | Ass Inst De Myologie | Tricyclo-DNA phosphorothioate |
| KR20140097398A (ko) | 2011-11-18 | 2014-08-06 | 사렙타 쎄러퓨틱스, 인코퍼레이티드 | 기능적으로-변형된 올리고뉴클레오티드 및 이의 서브유니트 |
| ES2727481T3 (es) * | 2011-11-30 | 2019-10-16 | Sarepta Therapeutics Inc | Inclusión inducida de exón en atrofia muscular espinal |
| CA2858576A1 (en) | 2011-12-06 | 2013-06-13 | Research Institute At Nationwide Children's Hospital | Non-ionic, low osmolar contrast agents for delivery of antisense oligonucleotides and treatment of disease |
| WO2013086444A2 (en) * | 2011-12-08 | 2013-06-13 | Sarepta Therapeutics, Inc. | Methods for treating progeroid laminopathies using oligonucleotide analogues targeting human lmna |
| CA2862628C (en) | 2012-01-27 | 2021-08-24 | Prosensa Technologies B.V. | Rna modulating oligonucleotides with improved characteristics for the treatment of duchenne and becker muscular dystrophy |
| WO2013173637A1 (en) | 2012-05-16 | 2013-11-21 | Rana Therapeutics, Inc. | Compositions and methods for modulating gene expression |
| EP2849800A4 (en) * | 2012-05-16 | 2015-12-09 | Rana Therapeutics Inc | COMPOUNDS AND METHOD FOR MODULATING BDNF EXPRESSION |
| HUE051612T2 (hu) | 2012-07-11 | 2021-03-01 | Sangamo Therapeutics Inc | Eljárások és készítmények lizoszomális tárolási betegségek kezelésére |
| AU2014317961B2 (en) | 2013-09-05 | 2020-07-30 | Murdoch University | Antisense-induced exon2 inclusion in acid alpha-glucosidase |
| US10724092B2 (en) | 2014-06-10 | 2020-07-28 | Erasmus University Medical Center Rotterdam | Methods for characterizing alternatively or aberrantly spliced mRNA isoforms |
| MX2017011004A (es) | 2015-02-27 | 2018-02-09 | Sarepta Therapeutics Inc | Inclusion del exon2 inducida por antisentido en alfa-glucosidasa.acida. |
| US10867398B2 (en) | 2017-11-21 | 2020-12-15 | Reliance Core Consulting LLC | Methods, systems, apparatuses and devices for facilitating motion analysis in an environment |
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