JP2019534298A5 - - Google Patents
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- Publication number
- JP2019534298A5 JP2019534298A5 JP2019523836A JP2019523836A JP2019534298A5 JP 2019534298 A5 JP2019534298 A5 JP 2019534298A5 JP 2019523836 A JP2019523836 A JP 2019523836A JP 2019523836 A JP2019523836 A JP 2019523836A JP 2019534298 A5 JP2019534298 A5 JP 2019534298A5
- Authority
- JP
- Japan
- Prior art keywords
- protein
- dux4
- composition according
- composition
- gene editing
- 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
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2023018459A JP2023071691A (ja) | 2016-11-07 | 2023-02-09 | 顔面肩甲上腕型筋ジストロフィーのための治療標的 |
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201662418694P | 2016-11-07 | 2016-11-07 | |
| US62/418,694 | 2016-11-07 | ||
| US201762490783P | 2017-04-27 | 2017-04-27 | |
| US62/490,783 | 2017-04-27 | ||
| PCT/US2017/060410 WO2018085842A1 (en) | 2016-11-07 | 2017-11-07 | Therapeutic targets for facioscapulohumeral muscular dystrophy |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023018459A Division JP2023071691A (ja) | 2016-11-07 | 2023-02-09 | 顔面肩甲上腕型筋ジストロフィーのための治療標的 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2019534298A JP2019534298A (ja) | 2019-11-28 |
| JP2019534298A5 true JP2019534298A5 (https=) | 2020-12-17 |
| JP7231226B2 JP7231226B2 (ja) | 2023-03-01 |
Family
ID=62076463
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019523836A Active JP7231226B2 (ja) | 2016-11-07 | 2017-11-07 | 顔面肩甲上腕型筋ジストロフィーのための治療標的 |
| JP2023018459A Pending JP2023071691A (ja) | 2016-11-07 | 2023-02-09 | 顔面肩甲上腕型筋ジストロフィーのための治療標的 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2023018459A Pending JP2023071691A (ja) | 2016-11-07 | 2023-02-09 | 顔面肩甲上腕型筋ジストロフィーのための治療標的 |
Country Status (6)
| Country | Link |
|---|---|
| US (3) | US11160823B2 (https=) |
| EP (2) | EP3896451B1 (https=) |
| JP (2) | JP7231226B2 (https=) |
| AU (1) | AU2017355712B2 (https=) |
| CA (1) | CA3042700A1 (https=) |
| WO (1) | WO2018085842A1 (https=) |
Families Citing this family (19)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US9228207B2 (en) | 2013-09-06 | 2016-01-05 | President And Fellows Of Harvard College | Switchable gRNAs comprising aptamers |
| US11160823B2 (en) | 2016-11-07 | 2021-11-02 | University Of Massachusetts | Therapeutic targets for facioscapulohumeral muscular dystrophy |
| EP3592853A1 (en) | 2017-03-09 | 2020-01-15 | President and Fellows of Harvard College | Suppression of pain by gene editing |
| US12390514B2 (en) | 2017-03-09 | 2025-08-19 | President And Fellows Of Harvard College | Cancer vaccine |
| CN111801345A (zh) | 2017-07-28 | 2020-10-20 | 哈佛大学的校长及成员们 | 使用噬菌体辅助连续进化(pace)的进化碱基编辑器的方法和组合物 |
| WO2019060432A2 (en) * | 2017-09-19 | 2019-03-28 | Children's National Medical Center | GAPMERS AND METHODS OF USING THE SAME FOR THE TREATMENT OF MUSCLE DYSTROPHY |
| KR102922694B1 (ko) | 2018-04-19 | 2026-02-03 | 더 리젠츠 오브 더 유니버시티 오브 캘리포니아 | 유전자 편집을 위한 조성물 및 방법 |
| US12522807B2 (en) | 2018-07-09 | 2026-01-13 | The Broad Institute, Inc. | RNA programmable epigenetic RNA modifiers and uses thereof |
| EP3906308A1 (en) * | 2018-12-31 | 2021-11-10 | Research Institute at Nationwide Children's Hospital | Dux4 rna silencing using rna targeting crispr-cas13b |
| US12351837B2 (en) | 2019-01-23 | 2025-07-08 | The Broad Institute, Inc. | Supernegatively charged proteins and uses thereof |
| WO2020191233A1 (en) | 2019-03-19 | 2020-09-24 | The Broad Institute, Inc. | Methods and compositions for editing nucleotide sequences |
| US12473543B2 (en) | 2019-04-17 | 2025-11-18 | The Broad Institute, Inc. | Adenine base editors with reduced off-target effects |
| WO2020252455A1 (en) * | 2019-06-13 | 2020-12-17 | The General Hospital Corporation | Engineered human-endogenous virus-like particles and methods of use thereof for delivery to cells |
| AU2021257213A1 (en) * | 2020-04-17 | 2022-11-03 | Nevada Research & Innovation Corporation | CRISPR-inhibition for facioscapulohumeral muscular dystrophy |
| CA3189601A1 (en) | 2020-07-24 | 2022-01-27 | The General Hospital Corporation | Enhanced virus-like particles and methods of use thereof for delivery to cells |
| WO2022045366A1 (en) * | 2020-08-31 | 2022-03-03 | Modalis Therapeutics Corporation | Method for treating facioscapulohumeral muscular dystrophy (fshd) by targeting dux4 gene |
| JP2022055361A (ja) * | 2020-09-28 | 2022-04-07 | 田辺三菱製薬株式会社 | Ｄｕｘ４の発現を調節するための医薬組成物 |
| CN120092093A (zh) * | 2022-08-25 | 2025-06-03 | 富士胶片株式会社 | 靶病毒的制造方法及培养组合物 |
| CN115896223A (zh) * | 2022-10-19 | 2023-04-04 | 中国医学科学院基础医学研究所 | 一种表观调节因子dot1l在肌肉分化中的应用 |
Family Cites Families (22)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US3854480A (en) | 1969-04-01 | 1974-12-17 | Alza Corp | Drug-delivery system |
| US4675189A (en) | 1980-11-18 | 1987-06-23 | Syntex (U.S.A.) Inc. | Microencapsulation of water soluble active polypeptides |
| US4452775A (en) | 1982-12-03 | 1984-06-05 | Syntex (U.S.A.) Inc. | Cholesterol matrix delivery system for sustained release of macromolecules |
| US5075109A (en) | 1986-10-24 | 1991-12-24 | Southern Research Institute | Method of potentiating an immune response |
| US5133974A (en) | 1989-05-05 | 1992-07-28 | Kv Pharmaceutical Company | Extended release pharmaceutical formulations |
| US5407686A (en) | 1991-11-27 | 1995-04-18 | Sidmak Laboratories, Inc. | Sustained release composition for oral administration of active ingredient |
| US6291438B1 (en) | 1993-02-24 | 2001-09-18 | Jui H. Wang | Antiviral anticancer poly-substituted phenyl derivatized oligoribonucleotides and methods for their use |
| US5858988A (en) | 1993-02-24 | 1999-01-12 | Wang; Jui H. | Poly-substituted-phenyl-oligoribo nucleotides having enhanced stability and membrane permeability and methods of use |
| US5736152A (en) | 1995-10-27 | 1998-04-07 | Atrix Laboratories, Inc. | Non-polymeric sustained release delivery system |
| US5684143A (en) | 1996-02-21 | 1997-11-04 | Lynx Therapeutics, Inc. | Oligo-2'-fluoronucleotide N3'->P5' phosphoramidates |
| WO2014071340A1 (en) * | 2012-11-05 | 2014-05-08 | University Of Washington Through Its Center For Commercialization | Methods and assays for facioscapulohumeral muscular dystrophy |
| WO2014144455A1 (en) | 2013-03-15 | 2014-09-18 | Epizyme, Inc. | 1 -phenoxy-3-(alkylamino)-propan-2-ol derivatives as carm1 inhibitors and uses thereof |
| CA2903813C (en) | 2013-03-15 | 2023-08-29 | Epizyme, Inc. | Carm1 inhibitors and uses thereof |
| US9814728B2 (en) * | 2013-09-20 | 2017-11-14 | Saint Louis University | Inhibition of DUX4 expression using bromodomain and extra-terminal domain protein inhibitors (BETi) |
| US20170283440A1 (en) | 2014-09-17 | 2017-10-05 | Epizyme, Inc. | Carm1 inhibitors and uses thereof |
| WO2016044604A1 (en) | 2014-09-17 | 2016-03-24 | Epizyme, Inc. | Carm1 inhibitors and uses thereof |
| WO2016044641A2 (en) | 2014-09-17 | 2016-03-24 | Epizyme, Inc. | Carm1 inhibitors and uses thereof |
| US10538763B2 (en) | 2015-01-16 | 2020-01-21 | Ionis Pharmaceuticals, Inc. | Compounds and methods for modulation of DUX4 |
| ES2926785T3 (es) * | 2015-06-02 | 2022-10-28 | Academisch Ziekenhuis Leiden | Medios y métodos para el tratamiento de la distrofia muscular facioescapulohumeral (FSHD) |
| WO2017201585A1 (en) | 2016-05-26 | 2017-11-30 | Genea Ip Holdings Pty Ltd | Modulators of dux4 for regulation of muscle function |
| EP3515506B1 (en) * | 2016-09-23 | 2023-09-13 | University of Massachusetts | Silencing of dux4 by recombinant gene editing complexes |
| US11160823B2 (en) | 2016-11-07 | 2021-11-02 | University Of Massachusetts | Therapeutic targets for facioscapulohumeral muscular dystrophy |
-
2017
- 2017-11-07 US US16/347,688 patent/US11160823B2/en active Active
- 2017-11-07 AU AU2017355712A patent/AU2017355712B2/en not_active Ceased
- 2017-11-07 JP JP2019523836A patent/JP7231226B2/ja active Active
- 2017-11-07 WO PCT/US2017/060410 patent/WO2018085842A1/en not_active Ceased
- 2017-11-07 EP EP21161592.7A patent/EP3896451B1/en active Active
- 2017-11-07 EP EP17867141.8A patent/EP3535588A4/en not_active Withdrawn
- 2017-11-07 CA CA3042700A patent/CA3042700A1/en active Pending
-
2021
- 2021-09-30 US US17/489,983 patent/US11963975B2/en active Active
-
2022
- 2022-04-06 US US17/714,349 patent/US20220226364A1/en not_active Abandoned
-
2023
- 2023-02-09 JP JP2023018459A patent/JP2023071691A/ja active Pending
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