JP2019515663A5 - - Google Patents
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- JP2019515663A5 JP2019515663A5 JP2018551837A JP2018551837A JP2019515663A5 JP 2019515663 A5 JP2019515663 A5 JP 2019515663A5 JP 2018551837 A JP2018551837 A JP 2018551837A JP 2018551837 A JP2018551837 A JP 2018551837A JP 2019515663 A5 JP2019515663 A5 JP 2019515663A5
- Authority
- JP
- Japan
- Prior art keywords
- mirna
- nucleic acid
- seq
- acid molecule
- composition
- 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.)
- Granted
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- 150000007523 nucleic acids Chemical class 0.000 claims description 42
- 108020004707 nucleic acids Proteins 0.000 claims description 40
- 102000039446 nucleic acids Human genes 0.000 claims description 40
- 239000000203 mixture Substances 0.000 claims description 36
- 241000702421 Dependoparvovirus Species 0.000 claims description 33
- 108091070501 miRNA Proteins 0.000 claims description 15
- 239000002679 microRNA Substances 0.000 claims description 15
- 239000003814 drug Substances 0.000 claims description 10
- 230000002401 inhibitory effect Effects 0.000 claims description 10
- 239000013598 vector Substances 0.000 claims description 10
- IQFYYKKMVGJFEH-XLPZGREQSA-N Thymidine Chemical group O=C1NC(=O)C(C)=CN1[C@@H]1O[C@H](CO)[C@@H](O)C1 IQFYYKKMVGJFEH-XLPZGREQSA-N 0.000 claims description 9
- OPTASPLRGRRNAP-UHFFFAOYSA-N cytosine Chemical compound NC=1C=CNC(=O)N=1 OPTASPLRGRRNAP-UHFFFAOYSA-N 0.000 claims description 9
- 238000002347 injection Methods 0.000 claims description 8
- 239000007924 injection Substances 0.000 claims description 8
- 238000002360 preparation method Methods 0.000 claims description 8
- 238000006467 substitution reaction Methods 0.000 claims description 8
- -1 miRNA-34 Proteins 0.000 claims description 7
- DWRXFEITVBNRMK-UHFFFAOYSA-N Beta-D-1-Arabinofuranosylthymine Natural products O=C1NC(=O)C(C)=CN1C1C(O)C(O)C(CO)O1 DWRXFEITVBNRMK-UHFFFAOYSA-N 0.000 claims description 6
- 241001465754 Metazoa Species 0.000 claims description 6
- IQFYYKKMVGJFEH-UHFFFAOYSA-N beta-L-thymidine Natural products O=C1NC(=O)C(C)=CN1C1OC(CO)C(O)C1 IQFYYKKMVGJFEH-UHFFFAOYSA-N 0.000 claims description 6
- 229940104302 cytosine Drugs 0.000 claims description 6
- 238000010255 intramuscular injection Methods 0.000 claims description 6
- 239000007927 intramuscular injection Substances 0.000 claims description 6
- 230000035800 maturation Effects 0.000 claims description 6
- 229940104230 thymidine Drugs 0.000 claims description 6
- 230000037317 transdermal delivery Effects 0.000 claims description 6
- 108020004414 DNA Proteins 0.000 claims description 5
- 101150054841 DUX4 gene Proteins 0.000 claims description 5
- 102100021158 Double homeobox protein 4 Human genes 0.000 claims description 5
- 101000968549 Homo sapiens Double homeobox protein 4 Proteins 0.000 claims description 5
- 230000001815 facial effect Effects 0.000 claims description 5
- 108090000623 proteins and genes Proteins 0.000 claims description 5
- 208000000943 scapulohumeral muscular dystrophy Diseases 0.000 claims description 5
- 210000002027 skeletal muscle Anatomy 0.000 claims description 5
- 241000700605 Viruses Species 0.000 claims description 3
- 241000972680 Adeno-associated virus - 6 Species 0.000 claims description 2
- 241000710929 Alphavirus Species 0.000 claims description 2
- 241000991587 Enterovirus C Species 0.000 claims description 2
- 241000283073 Equus caballus Species 0.000 claims description 2
- 241000713666 Lentivirus Species 0.000 claims description 2
- 108091028043 Nucleic acid sequence Proteins 0.000 claims description 2
- 241000710960 Sindbis virus Species 0.000 claims description 2
- 241000700618 Vaccinia virus Species 0.000 claims description 2
- 238000012217 deletion Methods 0.000 claims description 2
- 230000037430 deletion Effects 0.000 claims description 2
- 238000003780 insertion Methods 0.000 claims description 2
- 230000037431 insertion Effects 0.000 claims description 2
- 108091007423 let-7b Proteins 0.000 claims description 2
- 210000004185 liver Anatomy 0.000 claims description 2
- 108091029119 miR-34a stem-loop Proteins 0.000 claims description 2
- 108700000163 miRNA-29b Proteins 0.000 claims description 2
- 239000002773 nucleotide Substances 0.000 claims description 2
- 125000003729 nucleotide group Chemical group 0.000 claims description 2
- 239000013612 plasmid Substances 0.000 claims description 2
- 241000701161 unidentified adenovirus Species 0.000 claims description 2
- 241001529453 unidentified herpesvirus Species 0.000 claims description 2
- 241001430294 unidentified retrovirus Species 0.000 claims description 2
- 238000000034 method Methods 0.000 description 10
- NBQNWMBBSKPBAY-UHFFFAOYSA-N iodixanol Chemical compound IC=1C(C(=O)NCC(O)CO)=C(I)C(C(=O)NCC(O)CO)=C(I)C=1N(C(=O)C)CC(O)CN(C(C)=O)C1=C(I)C(C(=O)NCC(O)CO)=C(I)C(C(=O)NCC(O)CO)=C1I NBQNWMBBSKPBAY-UHFFFAOYSA-N 0.000 description 2
- 229960004359 iodixanol Drugs 0.000 description 2
- 238000000746 purification Methods 0.000 description 2
- 238000001190 Q-PCR Methods 0.000 description 1
- FAPWRFPIFSIZLT-UHFFFAOYSA-M Sodium chloride Chemical class [Na+].[Cl-] FAPWRFPIFSIZLT-UHFFFAOYSA-M 0.000 description 1
- 239000013024 dilution buffer Substances 0.000 description 1
- HQPMKSGTIOYHJT-UHFFFAOYSA-N ethane-1,2-diol;propane-1,2-diol Chemical compound OCCO.CC(O)CO HQPMKSGTIOYHJT-UHFFFAOYSA-N 0.000 description 1
- 239000006166 lysate Substances 0.000 description 1
- 229920001993 poloxamer 188 Polymers 0.000 description 1
- 238000004448 titration Methods 0.000 description 1
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2022005011A JP2022046792A (ja) | 2016-04-02 | 2022-01-17 | 組織特異的発現のための改変ｕ６プロモーターシステム |
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201662317524P | 2016-04-02 | 2016-04-02 | |
| US62/317,524 | 2016-04-02 | ||
| PCT/US2017/025614 WO2017173411A1 (en) | 2016-04-02 | 2017-03-31 | Modified u6 promoter system for tissue specific expression |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022005011A Division JP2022046792A (ja) | 2016-04-02 | 2022-01-17 | 組織特異的発現のための改変ｕ６プロモーターシステム |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2019515663A JP2019515663A (ja) | 2019-06-13 |
| JP2019515663A5 true JP2019515663A5 (https=) | 2020-04-30 |
| JP7253379B2 JP7253379B2 (ja) | 2023-04-06 |
Family
ID=59965277
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2018551837A Active JP7253379B2 (ja) | 2016-04-02 | 2017-03-31 | 組織特異的発現のための改変ｕ６プロモーターシステム |
| JP2022005011A Withdrawn JP2022046792A (ja) | 2016-04-02 | 2022-01-17 | 組織特異的発現のための改変ｕ６プロモーターシステム |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022005011A Withdrawn JP2022046792A (ja) | 2016-04-02 | 2022-01-17 | 組織特異的発現のための改変ｕ６プロモーターシステム |
Country Status (12)
| Country | Link |
|---|---|
| US (3) | US11345913B2 (https=) |
| EP (2) | EP4335502A3 (https=) |
| JP (2) | JP7253379B2 (https=) |
| KR (1) | KR102453187B1 (https=) |
| CN (1) | CN109714954A (https=) |
| AU (1) | AU2017240236C1 (https=) |
| BR (1) | BR112018070249A2 (https=) |
| CA (1) | CA3019832C (https=) |
| IL (1) | IL262056B2 (https=) |
| MA (1) | MA44528A (https=) |
| RU (1) | RU2768289C2 (https=) |
| WO (1) | WO2017173411A1 (https=) |
Families Citing this family (12)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| AU2018345772B2 (en) * | 2017-10-02 | 2025-04-17 | Research Institute At Nationwide Children's Hospital | MiRNA detargeting system for tissue specific interference |
| CN108823209B (zh) * | 2018-07-18 | 2021-04-09 | 华中农业大学 | 骨骼肌特异性启动子及应用 |
| SG11202100928QA (en) | 2018-08-02 | 2021-02-25 | Dyne Therapeutics Inc | Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy |
| US12018087B2 (en) | 2018-08-02 | 2024-06-25 | Dyne Therapeutics, Inc. | Muscle-targeting complexes comprising an anti-transferrin receptor antibody linked to an oligonucleotide and methods of delivering oligonucleotide to a subject |
| JP2021534755A (ja) | 2018-08-22 | 2021-12-16 | リサーチ インスティチュート アット ネイションワイド チルドレンズ ホスピタル | 筋強直性ジストロフィープロテインキナーゼの発現を抑制および／またはｄｍｐｋ遺伝子の３’非翻訳領域におけるトリヌクレオチドリピート伸長に干渉するための組換えウイルス産物および方法 |
| EP3906308A1 (en) * | 2018-12-31 | 2021-11-10 | Research Institute at Nationwide Children's Hospital | Dux4 rna silencing using rna targeting crispr-cas13b |
| KR20230023612A (ko) * | 2020-04-02 | 2023-02-17 | 마이레큘, 인크. | 조작된 올리고뉴클레오티드를 사용한 표적화된 억제 |
| CA3189065A1 (en) | 2020-09-11 | 2022-03-17 | Arrowhead Pharmaceuticals, Inc. | Rnai agents for inhibiting expression of dux4, compositions thereof, and methods of use |
| US12275941B2 (en) * | 2021-04-15 | 2025-04-15 | Research Institute At Nationwide Children's Hospital | Products and methods for inhibition of expression of dynamin-1 variants |
| EP4504952A4 (en) * | 2022-04-07 | 2026-04-08 | Kate Therapeutics Inc | COMPOSITIONS AND METHODS FOR MUSCULAR DISORDERS |
| WO2026011009A1 (en) | 2024-07-02 | 2026-01-08 | Kate Therapeutics, Inc. | Compositions and methods for muscle disorders |
| WO2026030242A1 (en) | 2024-07-29 | 2026-02-05 | Kate Therapeutics, Inc. | Adeno-associated virus compositions for the treatment of facioscapulohumeral muscular dystrophy |
Family Cites Families (33)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5173414A (en) | 1990-10-30 | 1992-12-22 | Applied Immune Sciences, Inc. | Production of recombinant adeno-associated virus vectors |
| WO1995013365A1 (en) | 1993-11-09 | 1995-05-18 | Targeted Genetics Corporation | Generation of high titers of recombinant aav vectors |
| PT728214E (pt) | 1993-11-09 | 2004-11-30 | Ohio Med College | Linhas celulares estaveis capazes de expressar o gene de replicacao do virus adeno-associado |
| US5658785A (en) | 1994-06-06 | 1997-08-19 | Children's Hospital, Inc. | Adeno-associated virus materials and methods |
| US5856152A (en) | 1994-10-28 | 1999-01-05 | The Trustees Of The University Of Pennsylvania | Hybrid adenovirus-AAV vector and methods of use therefor |
| CA2207927A1 (en) | 1994-12-06 | 1996-06-13 | Targeted Genetics Corporation | Packaging cell lines for generation of high titers of recombinant aav vectors |
| FR2737730B1 (fr) | 1995-08-10 | 1997-09-05 | Pasteur Merieux Serums Vacc | Procede de purification de virus par chromatographie |
| WO1997008298A1 (en) | 1995-08-30 | 1997-03-06 | Genzyme Corporation | Chromatographic purification of adenovirus and aav |
| US6632670B1 (en) | 1995-09-08 | 2003-10-14 | Genzyme Corporation | AAV vectors for gene therapy |
| US5910434A (en) | 1995-12-15 | 1999-06-08 | Systemix, Inc. | Method for obtaining retroviral packaging cell lines producing high transducing efficiency retroviral supernatant |
| JP2001500497A (ja) | 1996-09-06 | 2001-01-16 | トラステイーズ・オブ・ザ・ユニバーシテイ・オブ・ペンシルベニア | 組換えアデノ随伴ウイルスに指図される遺伝子治療の方法 |
| DK1009808T3 (da) | 1997-09-05 | 2013-01-21 | Genzyme Corp | Fremgangsmåder til generering af hjælper-frie præparater af rekombinante aav-vektorer med høj titer |
| US6566118B1 (en) | 1997-09-05 | 2003-05-20 | Targeted Genetics Corporation | Methods for generating high titer helper-free preparations of released recombinant AAV vectors |
| US6258595B1 (en) | 1999-03-18 | 2001-07-10 | The Trustees Of The University Of Pennsylvania | Compositions and methods for helper-free production of recombinant adeno-associated viruses |
| US7056502B2 (en) | 2000-04-28 | 2006-06-06 | The Trustees Of The University Of Pennsylvania | Recombinant aav vectors with AAV5 capsids and AAV5 vectors pseudotyped in heterologous capsids |
| WO2002053703A2 (en) | 2001-01-05 | 2002-07-11 | Children's Hospital, Inc. | Aav2 vectors and methods |
| PT1453547T (pt) * | 2001-12-17 | 2016-12-28 | Univ Pennsylvania | Sequências do vírus adeno-associado (aav) do serotipo 8, vetores contendo as mesmas, e utilizações destas |
| US8030473B2 (en) * | 2005-01-07 | 2011-10-04 | State Of Oregon Acting By And Through The State Board Of Higher Education On Behalf Of Oregon State University | Method to trigger RNA interference |
| DK2164967T3 (en) * | 2007-05-31 | 2015-10-19 | Univ Iowa Res Found | Reduction of off-target rna interferenstoksicitet |
| WO2009082488A2 (en) | 2007-12-24 | 2009-07-02 | Bergenbio As | Methods for creating and identifying functional rna interference elements |
| US9217155B2 (en) * | 2008-05-28 | 2015-12-22 | University Of Massachusetts | Isolation of novel AAV'S and uses thereof |
| EP3444346B1 (en) * | 2010-04-23 | 2022-07-27 | University of Massachusetts | Aav-based treatment of cholesterol-related disorders |
| ES2596977T3 (es) | 2010-09-02 | 2017-01-13 | Université de Mons | Agentes útiles en el tratamiento de la distrofia muscular facioescapulohumeral |
| WO2012143401A1 (en) * | 2011-04-18 | 2012-10-26 | INSERM (Institut National de la Santé et de la Recherche Médicale) | Tissue-specific regulation of transgene expression |
| EP2736539B1 (en) * | 2011-07-25 | 2017-08-23 | Nationwide Children's Hospital, Inc. | Recombinant virus products and methods for inhibition of expression of dux4 |
| WO2013120038A2 (en) * | 2012-02-10 | 2013-08-15 | Regents Of The University Of Minnesota | Morpholino targeting dux4 for treating fshd |
| DE102012007232B4 (de) | 2012-04-07 | 2014-03-13 | Susanne Weller | Verfahren zur Herstellung von rotierenden elektrischen Maschinen |
| CA2917348A1 (en) | 2013-07-11 | 2015-01-15 | Moderna Therapeutics, Inc. | Compositions comprising synthetic polynucleotides encoding crispr related proteins and synthetic sgrnas and methods of use |
| JP2015092462A (ja) | 2013-09-30 | 2015-05-14 | Tdk株式会社 | 正極及びそれを用いたリチウムイオン二次電池 |
| CA2942515C (en) | 2014-03-18 | 2025-12-09 | University Of Massachusetts | Raav-based compositions and methods for treating amyotrophic lateral sclerosis |
| WO2015141521A1 (ja) | 2014-03-21 | 2015-09-24 | 株式会社日立国際電気 | 基板処理装置、半導体装置の製造方法及び記録媒体 |
| EP3171895A1 (en) | 2014-07-23 | 2017-05-31 | Modernatx, Inc. | Modified polynucleotides for the production of intrabodies |
| JP6197169B2 (ja) | 2014-09-29 | 2017-09-20 | 東芝メモリ株式会社 | 半導体装置の製造方法 |
-
2017
- 2017-03-31 CA CA3019832A patent/CA3019832C/en active Active
- 2017-03-31 RU RU2018137677A patent/RU2768289C2/ru active
- 2017-03-31 EP EP23207092.0A patent/EP4335502A3/en active Pending
- 2017-03-31 BR BR112018070249A patent/BR112018070249A2/pt not_active Application Discontinuation
- 2017-03-31 EP EP17776877.7A patent/EP3435755B1/en active Active
- 2017-03-31 IL IL262056A patent/IL262056B2/en unknown
- 2017-03-31 CN CN201780033090.6A patent/CN109714954A/zh active Pending
- 2017-03-31 JP JP2018551837A patent/JP7253379B2/ja active Active
- 2017-03-31 KR KR1020187031571A patent/KR102453187B1/ko active Active
- 2017-03-31 WO PCT/US2017/025614 patent/WO2017173411A1/en not_active Ceased
- 2017-03-31 MA MA044528A patent/MA44528A/fr unknown
- 2017-03-31 US US16/089,860 patent/US11345913B2/en active Active
- 2017-03-31 AU AU2017240236A patent/AU2017240236C1/en active Active
-
2022
- 2022-01-17 JP JP2022005011A patent/JP2022046792A/ja not_active Withdrawn
- 2022-04-26 US US17/729,751 patent/US11939579B2/en active Active
-
2024
- 2024-02-16 US US18/444,111 patent/US20250019697A1/en not_active Abandoned
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