JPWO2019241624A5 - - Google Patents
Download PDFInfo
- Publication number
- JPWO2019241624A5 JPWO2019241624A5 JP2020568984A JP2020568984A JPWO2019241624A5 JP WO2019241624 A5 JPWO2019241624 A5 JP WO2019241624A5 JP 2020568984 A JP2020568984 A JP 2020568984A JP 2020568984 A JP2020568984 A JP 2020568984A JP WO2019241624 A5 JPWO2019241624 A5 JP WO2019241624A5
- Authority
- JP
- Japan
- Prior art keywords
- pharmaceutical composition
- composition according
- microrna
- promoter
- mir145
- 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
- 239000008194 pharmaceutical composition Substances 0.000 claims 31
- 229920001922 Mir-145 Polymers 0.000 claims 14
- 208000009575 Angelman Syndrome Diseases 0.000 claims 5
- 241000432074 Adeno-associated virus Species 0.000 claims 4
- 210000004556 Brain Anatomy 0.000 claims 4
- 239000004480 active ingredient Substances 0.000 claims 3
- 150000007523 nucleic acids Chemical class 0.000 claims 3
- 108020004707 nucleic acids Proteins 0.000 claims 3
- 241000702423 Adeno-associated virus - 2 Species 0.000 claims 2
- 102000004657 Calcium-Calmodulin-Dependent Protein Kinase Type 2 Human genes 0.000 claims 2
- 108010003721 Calcium-Calmodulin-Dependent Protein Kinase Type 2 Proteins 0.000 claims 2
- 108010065372 Dynorphins Proteins 0.000 claims 2
- 241000713666 Lentivirus Species 0.000 claims 2
- 241000283923 Marmota monax Species 0.000 claims 2
- 102100002857 PDYN Human genes 0.000 claims 2
- 102100007769 RIC8B Human genes 0.000 claims 2
- 101700025865 RIC8B Proteins 0.000 claims 2
- 102000017299 Synapsin-1 Human genes 0.000 claims 2
- 108050005241 Synapsin-1 Proteins 0.000 claims 2
- 230000001124 posttranscriptional Effects 0.000 claims 2
- 230000003612 virological Effects 0.000 claims 2
- 206010003591 Ataxia Diseases 0.000 claims 1
- 210000001218 Blood-Brain Barrier Anatomy 0.000 claims 1
- 206010010904 Convulsion Diseases 0.000 claims 1
- 206010012559 Developmental delay Diseases 0.000 claims 1
- 210000003414 Extremities Anatomy 0.000 claims 1
- 208000004141 Microcephaly Diseases 0.000 claims 1
- 206010039911 Seizure Diseases 0.000 claims 1
- 206010044565 Tremor Diseases 0.000 claims 1
- 108010006025 bovine growth hormone Proteins 0.000 claims 1
- 201000006347 intellectual disability Diseases 0.000 claims 1
- 239000002502 liposome Substances 0.000 claims 1
- 230000003137 locomotive Effects 0.000 claims 1
- 230000001404 mediated Effects 0.000 claims 1
- 239000000203 mixture Substances 0.000 claims 1
- 230000035699 permeability Effects 0.000 claims 1
- 239000008177 pharmaceutical agent Substances 0.000 claims 1
- 230000001402 polyadenylating Effects 0.000 claims 1
- 230000000699 topical Effects 0.000 claims 1
- 238000002604 ultrasonography Methods 0.000 claims 1
- XLYOFNOQVPJJNP-UHFFFAOYSA-N water Substances O XLYOFNOQVPJJNP-UHFFFAOYSA-N 0.000 claims 1
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201862684774P | 2018-06-14 | 2018-06-14 | |
| US62/684,774 | 2018-06-14 | ||
| PCT/US2019/037174 WO2019241624A1 (en) | 2018-06-14 | 2019-06-14 | Use of mir-92a or mir-145 in the treatment of angelman syndrome |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2021527087A JP2021527087A (ja) | 2021-10-11 |
| JPWO2019241624A5 true JPWO2019241624A5 (ko) | 2022-06-10 |
Family
ID=68839583
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020568984A Withdrawn JP2021527087A (ja) | 2018-06-14 | 2019-06-14 | アンジェルマン症候群の処置におけるＭＩＲ−９２ａまたはＭＩＲ−１４５の使用 |
Country Status (10)
| Country | Link |
|---|---|
| US (2) | US10704048B2 (ko) |
| EP (1) | EP3793567A4 (ko) |
| JP (1) | JP2021527087A (ko) |
| KR (1) | KR20210040358A (ko) |
| CN (1) | CN112739353A (ko) |
| AU (1) | AU2019285298A1 (ko) |
| CA (1) | CA3103267A1 (ko) |
| IL (1) | IL279391A (ko) |
| MX (1) | MX2020013667A (ko) |
| WO (1) | WO2019241624A1 (ko) |
Families Citing this family (3)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP2020533968A (ja) * | 2017-08-25 | 2020-11-26 | オービッド・セラピューティクス・インコーポレイテッドＯｖｉｄ Ｔｈｅｒａｐｅｕｔｉｃｓ Ｉｎｃ． | 組換えアデノ随伴ベクター |
| MX2020013667A (es) | 2018-06-14 | 2021-03-02 | Ovid Therapeutics Inc | Uso de mir-92a o mir-145 en el tratamiento del sindrome de angelman. |
| JP2023551911A (ja) * | 2020-12-01 | 2023-12-13 | ザ・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 | アンジェルマン症候群の治療のための組成物及びその使用 |
Family Cites Families (11)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5752515A (en) | 1996-08-21 | 1998-05-19 | Brigham & Women's Hospital | Methods and apparatus for image-guided ultrasound delivery of compounds through the blood-brain barrier |
| AU2006254732A1 (en) | 2005-06-03 | 2006-12-07 | Southern Adelaide Health Service-Flinders Medical Centre | Targeting cells with altered microrna expression |
| EP1937151A4 (en) | 2005-09-19 | 2011-07-06 | Univ Columbia | SYSTEMS AND METHOD FOR OPENING THE BLOOD-BRAIN BARRIER OF A PERSON WITH ULTRASOUND |
| CN102676516B (zh) * | 2011-03-17 | 2014-06-04 | 中国医学科学院肿瘤研究所 | microRNA 145的新用途 |
| WO2013056216A1 (en) * | 2011-10-14 | 2013-04-18 | New York University | Micrornas and methods of using same |
| EP2850190B1 (en) | 2012-05-16 | 2020-07-08 | Translate Bio MA, Inc. | Compositions and methods for modulating mecp2 expression |
| DK3461895T3 (da) | 2012-06-25 | 2020-07-20 | Ionis Pharmaceuticals Inc | Modulation af ube3a-ats-ekspression |
| ES2947311T3 (es) * | 2015-05-07 | 2023-08-04 | Univ South Florida | Gen UBE3A modificado para un enfoque de terapia génica para el síndrome de Angelman |
| US20190298843A1 (en) * | 2015-05-11 | 2019-10-03 | Purdue Research Foundation | Ligand ionophore conjugates |
| IL305342A (en) * | 2015-07-17 | 2023-10-01 | Ovid Therapeutics Inc | Methods for treating developmental disorders with gaboxadol |
| MX2020013667A (es) | 2018-06-14 | 2021-03-02 | Ovid Therapeutics Inc | Uso de mir-92a o mir-145 en el tratamiento del sindrome de angelman. |
-
2019
- 2019-06-14 MX MX2020013667A patent/MX2020013667A/es unknown
- 2019-06-14 US US16/441,391 patent/US10704048B2/en active Active
- 2019-06-14 AU AU2019285298A patent/AU2019285298A1/en not_active Abandoned
- 2019-06-14 WO PCT/US2019/037174 patent/WO2019241624A1/en unknown
- 2019-06-14 EP EP19819943.2A patent/EP3793567A4/en not_active Withdrawn
- 2019-06-14 CA CA3103267A patent/CA3103267A1/en active Pending
- 2019-06-14 KR KR1020217001109A patent/KR20210040358A/ko not_active Application Discontinuation
- 2019-06-14 CN CN201980040006.2A patent/CN112739353A/zh active Pending
- 2019-06-14 JP JP2020568984A patent/JP2021527087A/ja not_active Withdrawn
-
2020
- 2020-07-07 US US16/922,273 patent/US11130952B2/en active Active
- 2020-12-13 IL IL279391A patent/IL279391A/en unknown
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| RU2725813C2 (ru) | Векторы, содержащие спейсерные/филлер полинуклеотидные последовательности, и способы их применения | |
| RU2697444C2 (ru) | Вариант aav, композиции и способы, в которых он используется, а также способы его применения для переноса генов в клетки, органы и ткани | |
| US11344608B2 (en) | Factor IX gene therapy | |
| JP2021106619A5 (ko) | ||
| JP2021500049A (ja) | 神経変性疾患の遺伝子治療 | |
| JP2024095717A5 (ko) | ||
| US20140037585A1 (en) | AAV8 Vector with Enhanced Functional Activity and Methods of Use Thereof | |
| RU2020109343A (ru) | Полинуклеотиды aadc для лечения болезни паркинсона | |
| JP2020537542A (ja) | ライソゾーム病の遺伝子治療 | |
| JP2021502060A5 (ko) | ||
| KR20210096168A (ko) | 신경퇴행성 질환을 위한 유전자 요법 | |
| JP7541027B2 (ja) | 筋肉発現のためのハイブリッドプロモーター | |
| CN114231532B (zh) | 在哺乳动物肌肉中特异性启动基因的启动子序列及其应用 | |
| JP2023500793A (ja) | アルツハイマー病に対する遺伝子治療方法 | |
| US20230175015A1 (en) | Immunosuppressive agents and viral delivery re-dosing methods for gene therapy | |
| JPWO2019241624A5 (ko) | ||
| JP2020510433A5 (ko) | ||
| US20200283800A1 (en) | Gene therapies for neurodegenerative diseases | |
| JPWO2019153009A5 (ko) | ||
| JPWO2020069339A5 (ko) | ||
| JPWO2019210181A5 (ko) | ||
| JPWO2022053630A5 (ko) | ||
| JPWO2019152474A5 (ko) | ||
| JP2021505619A5 (ko) | ||
| JP2020533313A5 (ko) |