JP2020509786A5 - - Google Patents
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- Publication number
- JP2020509786A5 JP2020509786A5 JP2020500024A JP2020500024A JP2020509786A5 JP 2020509786 A5 JP2020509786 A5 JP 2020509786A5 JP 2020500024 A JP2020500024 A JP 2020500024A JP 2020500024 A JP2020500024 A JP 2020500024A JP 2020509786 A5 JP2020509786 A5 JP 2020509786A5
- Authority
- JP
- Japan
- Prior art keywords
- promoter
- nucleic acid
- seq
- group
- expression vector
- 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.)
- Pending
Links
- 150000007523 nucleic acids Chemical class 0.000 claims 24
- 239000013604 expression vector Substances 0.000 claims 13
- 241000702423 Adeno-associated virus - 2 Species 0.000 claims 12
- 108091028043 Nucleic acid sequence Proteins 0.000 claims 12
- 239000000203 mixture Substances 0.000 claims 10
- 241001634120 Adeno-associated virus - 5 Species 0.000 claims 8
- 241000972680 Adeno-associated virus - 6 Species 0.000 claims 8
- 241001164823 Adeno-associated virus - 7 Species 0.000 claims 8
- 241001164825 Adeno-associated virus - 8 Species 0.000 claims 8
- 241000701024 Human betaherpesvirus 5 Species 0.000 claims 8
- 108020004707 nucleic acids Proteins 0.000 claims 8
- 102000039446 nucleic acids Human genes 0.000 claims 8
- 239000013603 viral vector Substances 0.000 claims 8
- 210000004027 cell Anatomy 0.000 claims 7
- 101000798700 Homo sapiens Transmembrane protease serine 3 Proteins 0.000 claims 6
- 206010011878 Deafness Diseases 0.000 claims 5
- 101000798702 Homo sapiens Transmembrane protease serine 4 Proteins 0.000 claims 5
- 230000010370 hearing loss Effects 0.000 claims 5
- 231100000888 hearing loss Toxicity 0.000 claims 5
- 208000016354 hearing loss disease Diseases 0.000 claims 5
- 230000035772 mutation Effects 0.000 claims 5
- 239000008194 pharmaceutical composition Substances 0.000 claims 5
- 102000007469 Actins Human genes 0.000 claims 4
- 108010085238 Actins Proteins 0.000 claims 4
- 241001655883 Adeno-associated virus - 1 Species 0.000 claims 4
- 241000202702 Adeno-associated virus - 3 Species 0.000 claims 4
- 241000580270 Adeno-associated virus - 4 Species 0.000 claims 4
- 241000701022 Cytomegalovirus Species 0.000 claims 4
- 241000287828 Gallus gallus Species 0.000 claims 4
- 102000026889 Myosin VIIa Human genes 0.000 claims 4
- 108010009047 Myosin VIIa Proteins 0.000 claims 4
- 102100032454 Transmembrane protease serine 3 Human genes 0.000 claims 4
- 101150016977 pou4f3 gene Proteins 0.000 claims 4
- 210000001062 endolymphatic sac Anatomy 0.000 claims 2
- 230000002068 genetic effect Effects 0.000 claims 2
- 238000002347 injection Methods 0.000 claims 2
- 239000007924 injection Substances 0.000 claims 2
- 238000000034 method Methods 0.000 claims 2
- 210000000130 stem cell Anatomy 0.000 claims 2
- 101100343697 Homo sapiens LOXHD1 gene Proteins 0.000 claims 1
- 101001043326 Homo sapiens Lipoxygenase homology domain-containing protein 1 Proteins 0.000 claims 1
- 210000003027 ear inner Anatomy 0.000 claims 1
- 210000004263 induced pluripotent stem cell Anatomy 0.000 claims 1
- 230000002265 prevention Effects 0.000 claims 1
- 210000002480 semicircular canal Anatomy 0.000 claims 1
- 238000011830 transgenic mouse model Methods 0.000 claims 1
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201762472790P | 2017-03-17 | 2017-03-17 | |
| US62/472,790 | 2017-03-17 | ||
| US201762531522P | 2017-07-12 | 2017-07-12 | |
| US62/531,522 | 2017-07-12 | ||
| PCT/US2018/022873 WO2018170402A1 (en) | 2017-03-17 | 2018-03-16 | Gene therapy constructs and methods for treatment of hearing loss |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2020509786A JP2020509786A (ja) | 2020-04-02 |
| JP2020509786A5 true JP2020509786A5 (enExample) | 2021-03-11 |
Family
ID=63522674
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020500024A Pending JP2020509786A (ja) | 2017-03-17 | 2018-03-16 | 聴覚損失の治療のための遺伝子治療構成物および方法 |
Country Status (10)
| Country | Link |
|---|---|
| US (2) | US20200248203A1 (enExample) |
| EP (1) | EP3595634B1 (enExample) |
| JP (1) | JP2020509786A (enExample) |
| KR (1) | KR102752457B1 (enExample) |
| CN (1) | CN110709060A (enExample) |
| BR (1) | BR112019019367A2 (enExample) |
| CA (1) | CA3054941A1 (enExample) |
| ES (1) | ES2982270T3 (enExample) |
| IL (1) | IL268912B2 (enExample) |
| WO (1) | WO2018170402A1 (enExample) |
Families Citing this family (7)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US12391936B2 (en) | 2017-03-17 | 2025-08-19 | Rescue Hearing, Inc. | Gene therapy systems and related methods for treatment of hearing loss |
| CN115820654A (zh) * | 2019-08-30 | 2023-03-21 | 深圳华大基因股份有限公司 | Loxhd1基因突变体及其应用 |
| IL295265A (en) * | 2020-02-05 | 2022-10-01 | Rescue Hearing Inc | Gene therapy systems and related methods for treatment of hearing loss |
| EP4008788A1 (en) * | 2020-12-03 | 2022-06-08 | Medizinische Hochschule Hannover | Lentiviral vector technology for inner ear gene therapy |
| CN113584082B (zh) * | 2021-06-22 | 2023-07-25 | 复旦大学附属眼耳鼻喉科医院 | CRISPR/Cas9基因编辑系统及其在制备治疗遗传性感音神经性聋的药物中的应用 |
| CN113215248B (zh) * | 2021-06-25 | 2022-04-22 | 中国人民解放军空军军医大学 | 一种感音神经性耳聋相关的myo15a基因突变检测试剂盒 |
| WO2024259309A1 (en) * | 2023-06-15 | 2024-12-19 | Regeneron Pharmaceuticals, Inc. | Gene therapy for hearing disorders |
Family Cites Families (11)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP1280923A2 (en) * | 2000-04-28 | 2003-02-05 | Millennium Pharmaceuticals, Inc. | 14094, a human trypsin family member and uses thereof |
| WO2004099779A1 (en) * | 2003-05-05 | 2004-11-18 | Bayer Healthcare Ag | Diagnostics and therapeutics for diseases associated with human transmembrane serine protease 3 (tmprss3) |
| ZA200800970B (en) * | 2005-08-15 | 2009-10-28 | Genentech Inc | Gene disruptions, compositions and methods relating thereto |
| WO2011075838A1 (en) | 2009-12-21 | 2011-06-30 | Audigen Inc. | Method of treating or preventing hearing loss |
| US20150050354A1 (en) * | 2012-04-02 | 2015-02-19 | Moderna Therapeutics, Inc. | Modified polynucleotides for the treatment of otic diseases and conditions |
| EP2877213B1 (en) | 2012-07-25 | 2020-12-02 | The Broad Institute, Inc. | Inducible dna binding proteins and genome perturbation tools and applications thereof |
| US20160237455A1 (en) | 2013-09-27 | 2016-08-18 | Editas Medicine, Inc. | Crispr-related methods and compositions |
| JP6552511B2 (ja) | 2013-10-11 | 2019-07-31 | マサチューセッツ アイ アンド イヤー インファーマリー | 祖先ウイルス配列を予測する方法およびその使用 |
| WO2015175642A2 (en) * | 2014-05-13 | 2015-11-19 | Sangamo Biosciences, Inc. | Methods and compositions for prevention or treatment of a disease |
| WO2016069910A1 (en) | 2014-10-29 | 2016-05-06 | Massachusetts Eye And Ear Infirmary | Methods for efficient delivery of therapeutic molecules in vitro and in vivo |
| JP6990182B2 (ja) * | 2015-12-11 | 2022-02-15 | マサチューセッツ アイ アンド イヤー インファーマリー | 蝸牛および前庭細胞に核酸を送達するための材料および方法 |
-
2018
- 2018-03-16 KR KR1020197030012A patent/KR102752457B1/ko active Active
- 2018-03-16 BR BR112019019367A patent/BR112019019367A2/pt unknown
- 2018-03-16 WO PCT/US2018/022873 patent/WO2018170402A1/en not_active Ceased
- 2018-03-16 ES ES18768248T patent/ES2982270T3/es active Active
- 2018-03-16 US US16/488,103 patent/US20200248203A1/en active Pending
- 2018-03-16 CN CN201880028001.3A patent/CN110709060A/zh active Pending
- 2018-03-16 EP EP18768248.9A patent/EP3595634B1/en active Active
- 2018-03-16 IL IL268912A patent/IL268912B2/en unknown
- 2018-03-16 JP JP2020500024A patent/JP2020509786A/ja active Pending
- 2018-03-16 CA CA3054941A patent/CA3054941A1/en active Pending
-
2019
- 2019-12-24 US US16/726,495 patent/US20200114023A1/en active Pending
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