JP2018509154A5 - - Google Patents
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- Publication number
- JP2018509154A5 JP2018509154A5 JP2017547425A JP2017547425A JP2018509154A5 JP 2018509154 A5 JP2018509154 A5 JP 2018509154A5 JP 2017547425 A JP2017547425 A JP 2017547425A JP 2017547425 A JP2017547425 A JP 2017547425A JP 2018509154 A5 JP2018509154 A5 JP 2018509154A5
- Authority
- JP
- Japan
- Prior art keywords
- vector
- nucleotide sequence
- mammal
- motomeko
- sequence encoding
- 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
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- 239000013598 vector Substances 0.000 claims 27
- 239000003795 chemical substances by application Substances 0.000 claims 12
- 239000002773 nucleotide Substances 0.000 claims 9
- 125000003729 nucleotide group Chemical group 0.000 claims 9
- 241000124008 Mammalia Species 0.000 claims 8
- 239000012634 fragment Substances 0.000 claims 6
- KDCGOANMDULRCW-UHFFFAOYSA-N 7H-purine Chemical compound N1=CNC2=NC=NC2=C1 KDCGOANMDULRCW-UHFFFAOYSA-N 0.000 claims 4
- 101000899111 Homo sapiens Hemoglobin subunit beta Proteins 0.000 claims 4
- 102000004169 proteins and genes Human genes 0.000 claims 4
- 108090000623 proteins and genes Proteins 0.000 claims 4
- 210000001525 retina Anatomy 0.000 claims 4
- 241000701022 Cytomegalovirus Species 0.000 claims 3
- 239000013607 AAV vector Substances 0.000 claims 2
- 241000702423 Adeno-associated virus - 2 Species 0.000 claims 2
- 241000702421 Dependoparvovirus Species 0.000 claims 2
- 101100351286 Dictyostelium discoideum pdeE gene Proteins 0.000 claims 2
- 108091004242 G-Protein-Coupled Receptor Kinase 1 Proteins 0.000 claims 2
- 102000004437 G-Protein-Coupled Receptor Kinase 1 Human genes 0.000 claims 2
- 108090000799 Rhodopsin kinases Proteins 0.000 claims 2
- 108020004707 nucleic acids Proteins 0.000 claims 2
- 102000039446 nucleic acids Human genes 0.000 claims 2
- 150000007523 nucleic acids Chemical class 0.000 claims 2
- 101150037969 pde-6 gene Proteins 0.000 claims 2
- 108091008695 photoreceptors Proteins 0.000 claims 2
- 230000008488 polyadenylation Effects 0.000 claims 2
- 230000022532 regulation of transcription, DNA-dependent Effects 0.000 claims 2
- NCYCYZXNIZJOKI-IOUUIBBYSA-N 11-cis-retinal Chemical compound O=C/C=C(\C)/C=C\C=C(/C)\C=C\C1=C(C)CCCC1(C)C NCYCYZXNIZJOKI-IOUUIBBYSA-N 0.000 claims 1
- 241001164825 Adeno-associated virus - 8 Species 0.000 claims 1
- 108010003730 Cone Opsins Proteins 0.000 claims 1
- 108020004414 DNA Proteins 0.000 claims 1
- 108700024394 Exon Proteins 0.000 claims 1
- 101001104102 Homo sapiens X-linked retinitis pigmentosa GTPase regulator Proteins 0.000 claims 1
- 206010038848 Retinal detachment Diseases 0.000 claims 1
- 208000007014 Retinitis pigmentosa Diseases 0.000 claims 1
- 102000004330 Rhodopsin Human genes 0.000 claims 1
- 108090000820 Rhodopsin Proteins 0.000 claims 1
- 102100040092 X-linked retinitis pigmentosa GTPase regulator Human genes 0.000 claims 1
- 239000003937 drug carrier Substances 0.000 claims 1
- 239000000463 material Substances 0.000 claims 1
- 239000008194 pharmaceutical composition Substances 0.000 claims 1
- 230000002265 prevention Effects 0.000 claims 1
- 230000004264 retinal detachment Effects 0.000 claims 1
- 210000004358 rod cell outer segment Anatomy 0.000 claims 1
- 230000004304 visual acuity Effects 0.000 claims 1
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201562131661P | 2015-03-11 | 2015-03-11 | |
| US62/131,661 | 2015-03-11 | ||
| PCT/US2016/022072 WO2016145345A1 (en) | 2015-03-11 | 2016-03-11 | Rp2 and rpgr vectors for treating x-linked retinitis pigmentosa |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020167984A Division JP2021019611A (ja) | 2015-03-11 | 2020-10-02 | Ｘ連鎖性網膜色素変性症の治療のためのｒｐ２及びｒｐｇｒベクター |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2018509154A JP2018509154A (ja) | 2018-04-05 |
| JP2018509154A5 true JP2018509154A5 (enExample) | 2019-04-18 |
| JP6935049B2 JP6935049B2 (ja) | 2021-09-15 |
Family
ID=56879752
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2017547425A Active JP6935049B2 (ja) | 2015-03-11 | 2016-03-11 | Ｘ連鎖性網膜色素変性症の治療のためのｒｐ２及びｒｐｇｒベクター |
| JP2020167984A Pending JP2021019611A (ja) | 2015-03-11 | 2020-10-02 | Ｘ連鎖性網膜色素変性症の治療のためのｒｐ２及びｒｐｇｒベクター |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020167984A Pending JP2021019611A (ja) | 2015-03-11 | 2020-10-02 | Ｘ連鎖性網膜色素変性症の治療のためのｒｐ２及びｒｐｇｒベクター |
Country Status (6)
| Country | Link |
|---|---|
| US (2) | US10646588B2 (enExample) |
| EP (2) | EP3268481B1 (enExample) |
| JP (2) | JP6935049B2 (enExample) |
| AU (1) | AU2016228751B2 (enExample) |
| CA (1) | CA2979229A1 (enExample) |
| WO (1) | WO2016145345A1 (enExample) |
Families Citing this family (6)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| 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 |
| 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 |
| JOP20210054A1 (ar) * | 2018-09-21 | 2020-03-21 | Nightstarx Ltd | تركيبات وطرق لعلاج التهاب الشبكية الصباغي |
| WO2020172537A1 (en) * | 2019-02-22 | 2020-08-27 | University Of Massachusetts | Oxr1 gene therapy |
| WO2021041924A2 (en) * | 2019-08-30 | 2021-03-04 | Baylor College Of Medicine | System for regulating gene expression |
| WO2021178668A1 (en) * | 2020-03-04 | 2021-09-10 | University Of Massachusetts | Compositions and methods for modulating rpgr expression |
Family Cites Families (13)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| AU722375B2 (en) | 1996-09-06 | 2000-08-03 | Trustees Of The University Of Pennsylvania, The | Methods using cre-lox for production of recombinant adeno-associated viruses |
| ES2751999T3 (es) | 2008-01-29 | 2020-04-02 | Applied Genetic Tech Corporation | Producción recombinante de virus adeno-asociados usando células BHK en suspensión |
| WO2009105690A2 (en) * | 2008-02-21 | 2009-08-27 | Targeted Genetics Corporation | Devices and methods for delivering polynucleotides into retinal cells of the macula and fovea |
| US9868937B2 (en) | 2011-05-26 | 2018-01-16 | University Of Washington | Cell and gene based methods to improve cardiac function |
| 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 |
| US9873893B2 (en) * | 2013-02-15 | 2018-01-23 | The United States Of America As Represented By The Secretary, Dept. Of Health And Human Services | Methods and compositions for treating genetically linked diseases of the eye |
| EP2970972B1 (en) * | 2013-03-11 | 2018-08-15 | Fondazione Telethon | Mir-204 and mir-211 and uses thereof |
| JP6635942B2 (ja) | 2014-04-15 | 2020-01-29 | アプライド ジェネティック テクノロジーズ コーポレイション | 網膜色素変性症ｇｔｐアーゼレギュレーター（ｒｐｇｒ）をコードするコドン最適化核酸 |
| EA034355B1 (ru) * | 2014-05-02 | 2020-01-30 | Джензим Корпорейшн | Векторы на основе aav для генной терапии в сетчатке и cns |
| 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 |
| BR112017018728A2 (pt) * | 2015-03-03 | 2018-04-17 | Fond Telethon | sistema de vetor para expressar a sequência de codificação de um gene de interesse em uma célula, célula hospedeira, composição farmacêutica, método para tratar e/ou prevenir uma patologia ou doença, uso de uma sequência de nucleotídeo de um sinal de degradação em um sistema de vetor e método para diminuir a expressão de uma proteína na forma truncada |
| 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 |
-
2016
- 2016-03-11 CA CA2979229A patent/CA2979229A1/en active Pending
- 2016-03-11 EP EP16762623.3A patent/EP3268481B1/en active Active
- 2016-03-11 JP JP2017547425A patent/JP6935049B2/ja active Active
- 2016-03-11 WO PCT/US2016/022072 patent/WO2016145345A1/en not_active Ceased
- 2016-03-11 EP EP20176667.2A patent/EP3719134B1/en active Active
- 2016-03-11 US US15/556,746 patent/US10646588B2/en active Active
- 2016-03-11 AU AU2016228751A patent/AU2016228751B2/en active Active
-
2020
- 2020-04-21 US US16/854,605 patent/US11617801B2/en active Active
- 2020-10-02 JP JP2020167984A patent/JP2021019611A/ja active Pending
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