JP2017523239A5 - - Google Patents
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- 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 (2) | CN107206105A (enExample) |
| CA (1) | CA2991750C (enExample) |
| CY (1) | CY1123793T1 (enExample) |
| DK (2) | DK3821912T3 (enExample) |
| ES (2) | ES2987090T3 (enExample) |
| FI (1) | FI3821912T3 (enExample) |
| HR (2) | HRP20202023T1 (enExample) |
| HU (2) | HUE052781T2 (enExample) |
| LT (2) | LT3821912T (enExample) |
| PL (2) | PL3191139T3 (enExample) |
| PT (2) | PT3191139T (enExample) |
| RS (2) | RS65909B1 (enExample) |
| SI (2) | SI3821912T1 (enExample) |
| SM (2) | SMT202000715T1 (enExample) |
| WO (1) | WO2016014353A1 (enExample) |
Families Citing this family (12)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| SMT202000715T1 (it) * | 2014-07-24 | 2021-01-05 | Massachusetts Eye & Ear Infirmary | Terapia genica rpgr per la retinite pigmentosa |
| WO2016098078A2 (en) * | 2014-12-19 | 2016-06-23 | 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 |
| KR102763312B1 (ko) | 2017-10-20 | 2025-02-07 | 인쎄름 (엥스띠뛰 나씨오날 드 라 쌍떼 에 드 라 흐쉐르슈 메디깔) | 치료적으로 유효한 양의 재조합 aav9- 유도 벡터의 망막하 전달을 포함하는 대상체의 원추 광수용체 내 관심의 폴리뉴클레오티드를 발현하는 방법 |
| SMT202400321T1 (it) * | 2017-11-15 | 2024-11-15 | Univ Michigan Regents | Vettori virali comprendenti regioni codificanti rdh12 e metodi di trattamento di distrofie retiniche |
| MX2022011177A (es) | 2020-03-11 | 2022-12-13 | Massachusetts Eye & Ear Infirmary | Terapia genica para la degeneracion de la retina asociada a nmnat1. |
| CA3225080A1 (en) * | 2021-07-14 | 2023-01-19 | Anastasios GEORGIADIS | Kcnv2 gene therapy |
| CN117165596A (zh) * | 2022-05-27 | 2023-12-05 | 武汉纽福斯生物科技有限公司 | 编码rpgr的核酸及其应用 |
| 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 |
| WO2026150224A1 (en) | 2024-12-30 | 2026-07-16 | MeiraGTx Ocular UK Limited | Modified adeno-associated virus (aav) viral capsid proteins and methods of use |
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 |
| DE3681787D1 (de) | 1985-07-05 | 1991-11-07 | Whitehead Biomedical Inst | Expression von fremdem genetischem material in epithelzellen. |
| HK1008049A1 (en) | 1987-09-11 | 1999-04-30 | Whitehead Institute For Biomedical Research | Transduced fibroblasts and use therefor |
| ATE110108T1 (de) | 1987-12-11 | 1994-09-15 | Whitehead Biomedical Inst | Genetische modifizierung von endothelialen zellen. |
| DE68927996T2 (de) | 1988-02-05 | 1997-12-04 | Hughes Howard Med Inst | Modifizierte hepatozyten und deren anwendung |
| 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 |
| AU2044901A (en) | 1999-11-24 | 2001-06-04 | 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 |
| SMT202000715T1 (it) * | 2014-07-24 | 2021-01-05 | Massachusetts Eye & Ear Infirmary | Terapia genica rpgr per la retinite pigmentosa |
-
2015
- 2015-07-17 SM SM20200715T patent/SMT202000715T1/it unknown
- 2015-07-17 US US15/328,617 patent/US10314924B2/en active Active
- 2015-07-17 PL PL15825383T patent/PL3191139T3/pl unknown
- 2015-07-17 CN CN201580051512.3A patent/CN107206105A/zh active Pending
- 2015-07-17 HR HRP20202023TT patent/HRP20202023T1/hr unknown
- 2015-07-17 JP JP2017525514A patent/JP6654760B2/ja active Active
- 2015-07-17 SM SM20240309T patent/SMT202400309T1/it unknown
- 2015-07-17 CA CA2991750A patent/CA2991750C/en active Active
- 2015-07-17 HU HUE15825383A patent/HUE052781T2/hu unknown
- 2015-07-17 EP EP20199208.8A patent/EP3821912B1/en active Active
- 2015-07-17 EP EP15825383.1A patent/EP3191139B1/en active Active
- 2015-07-17 PT PT158253831T patent/PT3191139T/pt unknown
- 2015-07-17 DK DK20199208.8T patent/DK3821912T3/da active
- 2015-07-17 PT PT201992088T patent/PT3821912T/pt unknown
- 2015-07-17 WO PCT/US2015/040866 patent/WO2016014353A1/en not_active Ceased
- 2015-07-17 DK DK15825383.1T patent/DK3191139T3/da active
- 2015-07-17 LT LTEP20199208.8T patent/LT3821912T/lt unknown
- 2015-07-17 SI SI201532022T patent/SI3821912T1/sl unknown
- 2015-07-17 CN CN202511708312.2A patent/CN122235151A/zh active Pending
- 2015-07-17 RS RS20240901A patent/RS65909B1/sr unknown
- 2015-07-17 HU HUE20199208A patent/HUE068183T2/hu unknown
- 2015-07-17 ES ES20199208T patent/ES2987090T3/es active Active
- 2015-07-17 HR HRP20241154TT patent/HRP20241154T1/hr unknown
- 2015-07-17 PL PL20199208.8T patent/PL3821912T3/pl unknown
- 2015-07-17 LT LTEP15825383.1T patent/LT3191139T/lt unknown
- 2015-07-17 RS RS20201416A patent/RS61307B1/sr unknown
- 2015-07-17 FI FIEP20199208.8T patent/FI3821912T3/fi active
- 2015-07-17 SI SI201531413T patent/SI3191139T1/sl unknown
- 2015-07-17 ES ES15825383T patent/ES2834402T3/es active Active
-
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
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