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
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JP
Japan
Prior art keywords
vector
nucleotide sequence
mammal
motomeko
sequence encoding
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Application number
JP2017547425A
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English (en)
Japanese (ja)
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JP6935049B2 (ja
JP2018509154A (ja
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Priority claimed from PCT/US2016/022072 external-priority patent/WO2016145345A1/en
Publication of JP2018509154A publication Critical patent/JP2018509154A/ja
Publication of JP2018509154A5 publication Critical patent/JP2018509154A5/ja
Application granted granted Critical
Publication of JP6935049B2 publication Critical patent/JP6935049B2/ja
Active legal-status Critical Current
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JP2017547425A 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 Active JP6935049B2 (ja)

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) EP3719134B1 (enExample)
JP (2) JP6935049B2 (enExample)
AU (1) AU2016228751B2 (enExample)
CA (1) CA2979229A1 (enExample)
WO (1) WO2016145345A1 (enExample)

Families Citing this family (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2016228751B2 (en) * 2015-03-11 2022-01-13 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Rp2 vectors for treating x-linked retinitis pigmentosa
MY204271A (en) * 2017-11-15 2024-08-20 Ucl Business Ltd Viral vectors comprising rdh12 coding regions and methods of treating retinal dystrophies
JP2022501376A (ja) * 2018-09-21 2022-01-06 ナイトスター リミテッド 網膜色素変性症を処置するための組成物及び方法
WO2020172537A1 (en) * 2019-02-22 2020-08-27 University Of Massachusetts Oxr1 gene therapy
US20220290147A1 (en) * 2019-08-30 2022-09-15 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)

* Cited by examiner, † Cited by third party
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
CA2713338C (en) 2008-01-29 2021-10-26 Applied Genetic Technologies Corporation Recombinant virus production using mammalian cells in suspension
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
CA2837322C (en) 2011-05-26 2023-03-21 University Of Washington Cell and gene based methods to improve cardiac function
EP2872183B1 (en) * 2012-07-11 2018-09-26 The Trustees Of The University Of Pennsylvania Aav-mediated gene therapy for rpgr x-linked retinal degeneration
AU2014216160B2 (en) * 2013-02-15 2017-03-30 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services AAV8 retinoschisin expression vector for treating X-linked retinoschisis
CA2904164C (en) * 2013-03-11 2022-10-18 Fondazione Telethon Mir-204 and mir-211 and uses thereof
US9534225B2 (en) * 2014-04-15 2017-01-03 Applied Genetic Technologies Corporation Codon optimized nucleic acid encoding a retinitis pigmentosa gtpase regulator (RPGR)
EP3137497B2 (en) * 2014-05-02 2025-03-05 Genzyme Corporation Aav vectors for retinal and cns gene therapy
GB201412011D0 (en) * 2014-07-04 2014-08-20 Ucl Business Plc Treatments
WO2016014353A1 (en) * 2014-07-24 2016-01-28 Massachusetts Eye & Ear Infirmary Rpgr gene therapy for retinitis pigmentosa
US20180327779A1 (en) * 2015-03-03 2018-11-15 Fondazione Telethon (IT/IT) Multiple vector system and uses thereof
AU2016228751B2 (en) * 2015-03-11 2022-01-13 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Rp2 vectors for treating x-linked retinitis pigmentosa

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