JP2017523239A5 - - Google Patents
Download PDFInfo
- Publication number
- JP2017523239A5 JP2017523239A5 JP2017525514A JP2017525514A JP2017523239A5 JP 2017523239 A5 JP2017523239 A5 JP 2017523239A5 JP 2017525514 A JP2017525514 A JP 2017525514A JP 2017525514 A JP2017525514 A JP 2017525514A JP 2017523239 A5 JP2017523239 A5 JP 2017523239A5
- Authority
- JP
- Japan
- Prior art keywords
- seq
- nucleic acid
- protein
- rpgr
- human
- 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
- 101001104102 Homo sapiens X-linked retinitis pigmentosa GTPase regulator Proteins 0.000 claims description 40
- 102000039446 nucleic acids Human genes 0.000 claims description 26
- 108020004707 nucleic acids Proteins 0.000 claims description 26
- 150000007523 nucleic acids Chemical class 0.000 claims description 26
- 108090000623 proteins and genes Proteins 0.000 claims description 25
- 239000002299 complementary DNA Substances 0.000 claims description 23
- FWMNVWWHGCHHJJ-SKKKGAJSSA-N 4-amino-1-[(2r)-6-amino-2-[[(2r)-2-[[(2r)-2-[[(2r)-2-amino-3-phenylpropanoyl]amino]-3-phenylpropanoyl]amino]-4-methylpentanoyl]amino]hexanoyl]piperidine-4-carboxylic acid Chemical compound C([C@H](C(=O)N[C@H](CC(C)C)C(=O)N[C@H](CCCCN)C(=O)N1CCC(N)(CC1)C(O)=O)NC(=O)[C@H](N)CC=1C=CC=CC=1)C1=CC=CC=C1 FWMNVWWHGCHHJJ-SKKKGAJSSA-N 0.000 claims description 20
- 102100040092 X-linked retinitis pigmentosa GTPase regulator Human genes 0.000 claims description 20
- 102000050188 human RPGR Human genes 0.000 claims description 20
- 102000004169 proteins and genes Human genes 0.000 claims description 18
- 230000037430 deletion Effects 0.000 claims description 13
- 238000012217 deletion Methods 0.000 claims description 13
- 239000013603 viral vector Substances 0.000 claims description 13
- 208000007014 Retinitis pigmentosa Diseases 0.000 claims description 7
- 230000004777 loss-of-function mutation Effects 0.000 claims description 7
- 150000001413 amino acids Chemical class 0.000 claims description 6
- 241000702423 Adeno-associated virus - 2 Species 0.000 claims description 5
- 241000702421 Dependoparvovirus Species 0.000 claims description 5
- 239000013598 vector Substances 0.000 claims description 5
- 102000004437 G-Protein-Coupled Receptor Kinase 1 Human genes 0.000 claims description 4
- 101000611338 Homo sapiens Rhodopsin Proteins 0.000 claims description 4
- 206010025421 Macule Diseases 0.000 claims description 2
- 239000002773 nucleotide Substances 0.000 claims description 2
- 125000003729 nucleotide group Chemical group 0.000 claims description 2
- 210000001328 optic nerve Anatomy 0.000 claims description 2
- 239000008194 pharmaceutical composition Substances 0.000 claims 8
- 239000007788 liquid Substances 0.000 claims 1
- 238000000520 microinjection Methods 0.000 claims 1
- 238000000034 method Methods 0.000 description 9
- 239000012530 fluid Substances 0.000 description 1
- 238000012986 modification Methods 0.000 description 1
- 230000004048 modification Effects 0.000 description 1
Priority Applications (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2019235539A JP6966532B2 (ja) | 2014-07-24 | 2019-12-26 | 網膜色素変性症のためのｒｐｇｒ遺伝子治療 |
| JP2021172128A JP7198329B2 (ja) | 2014-07-24 | 2021-10-21 | 網膜色素変性症のためのｒｐｇｒ遺伝子治療 |
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201462028638P | 2014-07-24 | 2014-07-24 | |
| US62/028,638 | 2014-07-24 | ||
| PCT/US2015/040866 WO2016014353A1 (en) | 2014-07-24 | 2015-07-17 | Rpgr gene therapy for retinitis pigmentosa |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019235539A Division JP6966532B2 (ja) | 2014-07-24 | 2019-12-26 | 網膜色素変性症のためのｒｐｇｒ遺伝子治療 |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2017523239A JP2017523239A (ja) | 2017-08-17 |
| JP2017523239A5 true JP2017523239A5 (enExample) | 2018-08-16 |
| JP6654760B2 JP6654760B2 (ja) | 2020-02-26 |
Family
ID=55163577
Family Applications (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2017525514A Active JP6654760B2 (ja) | 2014-07-24 | 2015-07-17 | 網膜色素変性症のためのｒｐｇｒ遺伝子治療 |
| JP2019235539A Active JP6966532B2 (ja) | 2014-07-24 | 2019-12-26 | 網膜色素変性症のためのｒｐｇｒ遺伝子治療 |
| JP2021172128A Active JP7198329B2 (ja) | 2014-07-24 | 2021-10-21 | 網膜色素変性症のためのｒｐｇｒ遺伝子治療 |
Family Applications After (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019235539A Active JP6966532B2 (ja) | 2014-07-24 | 2019-12-26 | 網膜色素変性症のためのｒｐｇｒ遺伝子治療 |
| JP2021172128A Active JP7198329B2 (ja) | 2014-07-24 | 2021-10-21 | 網膜色素変性症のためのｒｐｇｒ遺伝子治療 |
Country Status (18)
| Country | Link |
|---|---|
| US (2) | US10314924B2 (enExample) |
| EP (2) | EP3821912B1 (enExample) |
| JP (3) | JP6654760B2 (enExample) |
| CN (1) | CN107206105A (enExample) |
| CA (1) | CA2991750C (enExample) |
| CY (1) | CY1123793T1 (enExample) |
| DK (2) | DK3191139T3 (enExample) |
| ES (2) | ES2834402T3 (enExample) |
| FI (1) | FI3821912T3 (enExample) |
| HR (2) | HRP20241154T1 (enExample) |
| HU (2) | HUE068183T2 (enExample) |
| LT (2) | LT3191139T (enExample) |
| PL (2) | PL3821912T3 (enExample) |
| PT (2) | PT3191139T (enExample) |
| RS (2) | RS61307B1 (enExample) |
| SI (2) | SI3191139T1 (enExample) |
| SM (2) | SMT202000715T1 (enExample) |
| WO (1) | WO2016014353A1 (enExample) |
Families Citing this family (9)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| ES2834402T3 (es) * | 2014-07-24 | 2021-06-17 | Massachusetts Eye & Ear Infirmary | Terapia génica con RPGR para retinitis pigmentosa |
| US20190054117A1 (en) * | 2014-12-19 | 2019-02-21 | Novartis Ag | Dimerization switches and uses thereof |
| CA2979229A1 (en) * | 2015-03-11 | 2016-09-15 | The United States Of America, As Represented By The Secretary, Department Of Health And Human Services | Rp2 vectors for treating x-linked retinitis pigmentosa |
| GB201516066D0 (en) | 2015-09-10 | 2015-10-28 | Young & Co Llp D | Treatment of retinitis pigmentosa |
| GB201704192D0 (en) | 2017-03-16 | 2017-05-03 | Nightstarx Ltd | Treatment of Retinitis Pigmentosa |
| EP3697448A1 (en) * | 2017-10-20 | 2020-08-26 | INSERM (Institut National de la Santé et de la Recherche Médicale) | Methods of expressing a polynucleotide of interest in the cone photoreceptors of a subject comprising the subretinal delivery of a therapeutically effective amount of a recombinant aav9-derived vector |
| SI3710590T1 (sl) * | 2017-11-15 | 2024-10-30 | The Regents Of The University Of Michigan | Virusni vektorji, ki obsegajo kodirne regije RDH12 in postopke zdravljenja retinalnih distrofij |
| MX2022011177A (es) * | 2020-03-11 | 2022-12-13 | Massachusetts Eye & Ear Infirmary | Terapia genica para la degeneracion de la retina asociada a nmnat1. |
| CN120350069A (zh) * | 2024-01-12 | 2025-07-22 | 北京中因科技有限公司 | 视网膜色素变性动物模型及其构建方法和应用 |
Family Cites Families (13)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US4980286A (en) | 1985-07-05 | 1990-12-25 | Whitehead Institute For Biomedical Research | In vivo introduction and expression of foreign genetic material in epithelial cells |
| EP0228458B2 (en) | 1985-07-05 | 1997-10-22 | Whitehead Institute For Biomedical Research | Epithelial cells expressing foreign genetic material |
| ATE117375T1 (de) | 1987-09-11 | 1995-02-15 | Whitehead Biomedical Inst | Transduktionsveränderte fibroblasten und ihre anwendung. |
| EP0391960B1 (en) | 1987-12-11 | 1994-08-17 | Whitehead Institute For Biomedical Research | Genetic modification of endothelial cells |
| JP2917998B2 (ja) | 1988-02-05 | 1999-07-12 | ホワイトヘッド・インスティチュート・フォー・バイオメディカル・リサーチ | 修飾された肝細胞およびその用途 |
| DE69123981T3 (de) | 1990-10-31 | 2006-06-14 | Cell Genesys Inc | Nützliche retrovirale vektoren für die gentherapie |
| US5767079A (en) * | 1992-07-08 | 1998-06-16 | Celtrix Pharmaceuticals, Inc. | Method of treating ophthalmic disorders using TGF -β |
| WO2001038578A1 (en) * | 1999-11-24 | 2001-05-31 | Cornell Research Foundation, Inc. | Inherited retinal diseases at the canine rp3 locus: linkage, marker- and mutation-based tests |
| GB0008801D0 (en) * | 2000-04-10 | 2000-05-31 | Medical Res Council | Sequences |
| CN102925445A (zh) * | 2011-08-08 | 2013-02-13 | 刘军 | 一种获得rpgr 基因新的转录剪切形式的方法 |
| CN105120901A (zh) * | 2012-07-11 | 2015-12-02 | 宾夕法尼亚大学托管会 | Rpgr x染色体连锁视网膜退化的aav介导的基因治疗 |
| GB201412011D0 (en) * | 2014-07-04 | 2014-08-20 | Ucl Business Plc | Treatments |
| ES2834402T3 (es) * | 2014-07-24 | 2021-06-17 | Massachusetts Eye & Ear Infirmary | Terapia génica con RPGR para retinitis pigmentosa |
-
2015
- 2015-07-17 ES ES15825383T patent/ES2834402T3/es active Active
- 2015-07-17 HU HUE20199208A patent/HUE068183T2/hu unknown
- 2015-07-17 CA CA2991750A patent/CA2991750C/en active Active
- 2015-07-17 SI SI201531413T patent/SI3191139T1/sl unknown
- 2015-07-17 US US15/328,617 patent/US10314924B2/en active Active
- 2015-07-17 SI SI201532022T patent/SI3821912T1/sl unknown
- 2015-07-17 PT PT158253831T patent/PT3191139T/pt unknown
- 2015-07-17 PT PT201992088T patent/PT3821912T/pt unknown
- 2015-07-17 JP JP2017525514A patent/JP6654760B2/ja active Active
- 2015-07-17 WO PCT/US2015/040866 patent/WO2016014353A1/en not_active Ceased
- 2015-07-17 EP EP20199208.8A patent/EP3821912B1/en active Active
- 2015-07-17 HR HRP20241154TT patent/HRP20241154T1/hr unknown
- 2015-07-17 PL PL20199208.8T patent/PL3821912T3/pl unknown
- 2015-07-17 ES ES20199208T patent/ES2987090T3/es active Active
- 2015-07-17 DK DK15825383.1T patent/DK3191139T3/da active
- 2015-07-17 LT LTEP15825383.1T patent/LT3191139T/lt unknown
- 2015-07-17 RS RS20201416A patent/RS61307B1/sr unknown
- 2015-07-17 LT LTEP20199208.8T patent/LT3821912T/lt unknown
- 2015-07-17 FI FIEP20199208.8T patent/FI3821912T3/fi active
- 2015-07-17 HR HRP20202023TT patent/HRP20202023T1/hr unknown
- 2015-07-17 SM SM20200715T patent/SMT202000715T1/it unknown
- 2015-07-17 DK DK20199208.8T patent/DK3821912T3/da active
- 2015-07-17 PL PL15825383T patent/PL3191139T3/pl unknown
- 2015-07-17 EP EP15825383.1A patent/EP3191139B1/en active Active
- 2015-07-17 RS RS20240901A patent/RS65909B1/sr unknown
- 2015-07-17 SM SM20240309T patent/SMT202400309T1/it unknown
- 2015-07-17 HU HUE15825383A patent/HUE052781T2/hu unknown
- 2015-07-17 CN CN201580051512.3A patent/CN107206105A/zh active Pending
-
2019
- 2019-06-10 US US16/436,521 patent/US11045558B2/en active Active
- 2019-12-26 JP JP2019235539A patent/JP6966532B2/ja active Active
-
2021
- 2021-01-05 CY CY20211100002T patent/CY1123793T1/el unknown
- 2021-10-21 JP JP2021172128A patent/JP7198329B2/ja active Active
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| JP2017523239A5 (enExample) | ||
| JP2020073536A5 (enExample) | ||
| US12180500B2 (en) | Chimeric capsids | |
| AU2013287281B2 (en) | AAV-mediated gene therapy for RPGR x-linked retinal degeneration | |
| HRP20202023T1 (hr) | Genska terapija rpgr za liječenje retinitis pigmentose | |
| JP2015508760A5 (enExample) | ||
| JP2017510296A5 (enExample) | ||
| JP2018520646A5 (enExample) | ||
| KR20210028162A (ko) | 지대근 이영양증 2a형을 치료하기 위한 재조합 아데노 연관 바이러스 생성물 및 방법 | |
| JP2014518614A (ja) | 変異体キャプシドを有するアデノ関連ウイルスビリオンおよびその使用方法 | |
| JP2019537437A5 (enExample) | ||
| JP2016535034A5 (enExample) | ||
| CN104936606A (zh) | 糖原贮积病的基因疗法 | |
| JP2021502978A5 (enExample) | ||
| IL308645A (en) | Viral vectors comprising rdh12 coding regions and methods of treating retinal dystrophies | |
| BR112021012048A2 (pt) | Fator complementar i e cofator de fator complementar i, vetores que codificam os mesmos e uso terapêutico | |
| JP2019524090A5 (enExample) | ||
| RU2019126509A (ru) | Выделенный модифицированный белок VP1 капсида аденоассоциированного вируса 5 серотипа (AAV5), капсид и вектор на его основе | |
| McClements et al. | Assessment of AAV dual vector safety in the Abca4−/− mouse model of Stargardt disease | |
| JP2018526994A5 (enExample) | ||
| JP2018509154A5 (enExample) | ||
| JP2021520232A (ja) | 黄斑ジストロフィーを処置するための組成物及び方法 | |
| HK1253299A1 (zh) | 一种通过基因治疗治疗眼疾病的改进方法 | |
| US20170137480A1 (en) | Rpgr-ofr15 variant gene in the treatment of retinitis pigmentosa | |
| Vandenberghe | What is next for retinal gene therapy? |