JP2019511916A5 - - Google Patents
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- Publication number
- JP2019511916A5 JP2019511916A5 JP2018544786A JP2018544786A JP2019511916A5 JP 2019511916 A5 JP2019511916 A5 JP 2019511916A5 JP 2018544786 A JP2018544786 A JP 2018544786A JP 2018544786 A JP2018544786 A JP 2018544786A JP 2019511916 A5 JP2019511916 A5 JP 2019511916A5
- Authority
- JP
- Japan
- Prior art keywords
- dux4
- composition
- associated virus
- construct
- 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.)
- Granted
Links
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201662300500P | 2016-02-26 | 2016-02-26 | |
| US62/300,500 | 2016-02-26 | ||
| PCT/US2017/019422 WO2017147467A1 (en) | 2016-02-26 | 2017-02-24 | Recombinant virus products and methods for inducing dux4 exon skipping |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2019511916A JP2019511916A (ja) | 2019-05-09 |
| JP2019511916A5 true JP2019511916A5 (https=) | 2020-06-11 |
| JP6966463B2 JP6966463B2 (ja) | 2021-11-17 |
Family
ID=59685610
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2018544786A Active JP6966463B2 (ja) | 2016-02-26 | 2017-02-24 | 組換えウイルス産物及びｄｕｘ４エクソンスキッピングを誘導するための方法 |
Country Status (5)
| Country | Link |
|---|---|
| US (2) | US11180755B2 (https=) |
| EP (1) | EP3420109A4 (https=) |
| JP (1) | JP6966463B2 (https=) |
| AU (2) | AU2017222653B2 (https=) |
| WO (1) | WO2017147467A1 (https=) |
Families Citing this family (8)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2019051290A1 (en) * | 2017-09-07 | 2019-03-14 | The Children's Medical Center Corporation | COMPOSITIONS AND METHODS FOR THE TREATMENT OF FACIO-SCAPULO-HUMERAL DYSTROPHY |
| EP3906308A1 (en) * | 2018-12-31 | 2021-11-10 | Research Institute at Nationwide Children's Hospital | Dux4 rna silencing using rna targeting crispr-cas13b |
| US20220364086A1 (en) * | 2019-07-12 | 2022-11-17 | Daiichi Sankyo Company, Limited | ANTISENSE OLIGONUCLEOTIDE CAPABLE OF ALTERING SPLICING OF DUX4 pre-mRNA |
| IL303230A (en) * | 2020-11-30 | 2023-07-01 | Res Inst Nationwide Childrens Hospital | Compositions and methods for treating facioscapulohumeral muscular dystrophy (fshd) |
| WO2022240760A2 (en) | 2021-05-10 | 2022-11-17 | Entrada Therapeutics, Inc. | COMPOSITIONS AND METHODS FOR MODULATING mRNA SPLICING |
| AU2023228712A1 (en) | 2022-03-04 | 2024-10-17 | Regeneron Pharmaceuticals, Inc. | Compositions and methods comprising engineered small nuclear rna (snrna) |
| 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 (25)
| 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 |
| 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 |
| WO1995013365A1 (en) | 1993-11-09 | 1995-05-18 | Targeted Genetics Corporation | Generation of high titers of recombinant aav vectors |
| 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 |
| US6566118B1 (en) | 1997-09-05 | 2003-05-20 | Targeted Genetics Corporation | Methods for generating high titer helper-free preparations of released recombinant AAV vectors |
| DK1009808T3 (da) | 1997-09-05 | 2013-01-21 | Genzyme Corp | Fremgangsmåder til generering af hjælper-frie præparater af rekombinante aav-vektorer med høj titer |
| 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 |
| US9080170B2 (en) | 2010-03-17 | 2015-07-14 | Association Institut De Myologie | Modified U7 snRNAs for treatment of neuromuscular diseases |
| ES2596977T3 (es) | 2010-09-02 | 2017-01-13 | Université de Mons | Agentes útiles en el tratamiento de la distrofia muscular facioescapulohumeral |
| EP2500434A1 (en) | 2011-03-12 | 2012-09-19 | Association Institut de Myologie | Capsid-free AAV vectors, compositions, and methods for vector production and gene delivery |
| 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 |
| 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 |
| 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 |
| 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
- 2017-02-24 JP JP2018544786A patent/JP6966463B2/ja active Active
- 2017-02-24 EP EP17757330.0A patent/EP3420109A4/en active Pending
- 2017-02-24 AU AU2017222653A patent/AU2017222653B2/en active Active
- 2017-02-24 US US16/079,426 patent/US11180755B2/en active Active
- 2017-02-24 WO PCT/US2017/019422 patent/WO2017147467A1/en not_active Ceased
-
2021
- 2021-10-13 US US17/500,410 patent/US12084658B2/en active Active
-
2023
- 2023-08-14 AU AU2023216733A patent/AU2023216733B2/en active Active
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