JP2018509154A5 - - Google Patents

Download PDF

Info

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
Application number
JP2017547425A
Other languages
English (en)
Japanese (ja)
Other versions
JP6935049B2 (ja
JP2018509154A (ja
Filing date
Publication date
Application filed filed Critical
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
Anticipated expiration legal-status Critical

Links

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 (9)

* Cited by examiner, † Cited by third party
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
SMT202400321T1 (it) * 2017-11-15 2024-11-15 Univ Michigan Regents Vettori virali comprendenti regioni codificanti rdh12 e metodi di trattamento di distrofie retiniche
TW202028225A (zh) * 2018-09-21 2020-08-01 英商夜星克有限公司 用於治療色素性視網膜炎之組合物及方法
CA3131023A1 (en) * 2019-02-22 2020-08-27 Michael R. Volkert Oxr1 gene therapy
KR20220049619A (ko) * 2019-08-30 2022-04-21 베이롤 칼리지 오브 메드신 유전자 발현 조절 시스템
WO2021178668A1 (en) * 2020-03-04 2021-09-10 University Of Massachusetts Compositions and methods for modulating rpgr expression
EP4511496A4 (en) * 2022-04-18 2026-06-24 Abeona Therapeutics Inc COMPOSITIONS AND METHODS FOR THE TREATMENT OF DOMINANT OPTIC ATROPHY AND X-RELATED RETINOSCHISISIS
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
WO2026119796A1 (en) * 2024-12-05 2026-06-11 Beacon Therapeutics Limited Gene therapy vector

Family Cites Families (13)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1998010086A1 (en) 1996-09-06 1998-03-12 Trustees Of The University Of Pennsylvania Methods using cre-lox for production of recombinant adeno-associated viruses
EP2242840B1 (en) 2008-01-29 2019-07-24 Applied Genetic Technologies Corporation Recombinant adeno-associated virus production using bhk cells in suspension
US20100081707A1 (en) * 2008-02-21 2010-04-01 Ali Robin R 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
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
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
CN105209619B (zh) * 2013-03-11 2018-10-12 泰莱托恩基金会 miR-204和miR-211及其用途
EP3132051B1 (en) * 2014-04-15 2019-03-20 Applied Genetic Technologies Corporation Codon optimized nucleic acid encoding a retinitis pigmentosa gtpase regulator (rpgr)
LT3137497T (lt) * 2014-05-02 2021-07-26 Genzyme Corporation Aav vektoriai, skirti tinklainės ir cns genų terapijai
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
CA2979120A1 (en) * 2015-03-03 2016-09-09 Fondazione Telethon Multiple aav vector system for expressing genes over 5 kilobases, comprising at least one degradation signal 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

Similar Documents

Publication Publication Date Title
JP6831779B2 (ja) 修飾された第ix因子、並びに、細胞、器官及び組織への遺伝子導入のための組成物、方法及び使用
US20220331409A1 (en) Factor ix gene therapy
JP2020514286A5 (enExample)
JP6805174B2 (ja) 神経成長因子シグナルペプチド及び副甲状腺ホルモンを含むaav分離株及び融合タンパク質
JP2016538885A5 (enExample)
JP2015523379A5 (enExample)
JP2014512171A5 (enExample)
JP2019523648A5 (enExample)
RU2019100525A (ru) Векторная система на основе аденоассоциированного вируса
JP2018537984A5 (enExample)
JP2019524090A5 (enExample)
JP6767483B2 (ja) 遺伝子療法のための、改変されたフリードライヒ運動失調症遺伝子およびベクター
JP2020073536A5 (enExample)
CA3024449A1 (en) Compositions and methods of treating huntington's disease
JP2019502378A5 (enExample)
KR20160033217A (ko) 변종 aav 및 조성물, 세포, 기관 및 조직으로의 유전자 전이를 위한 방법 및 용도
KR20170084012A (ko) 지혈 장애의 치료를 위한 변이체 인자 viii의 패키징 및 발현을 위한 개선된 발현 카세트
JP2016500519A5 (enExample)
JP2018510620A5 (enExample)
JP2023528801A5 (enExample)
JP2017523239A5 (enExample)
JP2021500922A5 (enExample)
JP2022529782A (ja) シュタルガルト病(abca4)の遺伝子治療
Hollinger et al. Viral vector mediated gene therapies
JP2021502978A5 (enExample)