KR102612871B1 - 마이오실린(myoc) 녹내장을 치료하기 위한 아데노-관련 바이러스 벡터 - Google Patents

마이오실린(myoc) 녹내장을 치료하기 위한 아데노-관련 바이러스 벡터 Download PDF

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KR102612871B1
KR102612871B1 KR1020177010012A KR20177010012A KR102612871B1 KR 102612871 B1 KR102612871 B1 KR 102612871B1 KR 1020177010012 A KR1020177010012 A KR 1020177010012A KR 20177010012 A KR20177010012 A KR 20177010012A KR 102612871 B1 KR102612871 B1 KR 102612871B1
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myoc
raav
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KR20170054493A (ko
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피터 퍼찬
에이브러햄 스카리아
제프리 아딘저
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젠자임 코포레이션
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KR1020177010012A 2014-09-16 2015-09-16 마이오실린(myoc) 녹내장을 치료하기 위한 아데노-관련 바이러스 벡터 Active KR102612871B1 (ko)

Priority Applications (1)

Application Number Priority Date Filing Date Title
KR1020237042363A KR20230172610A (ko) 2014-09-16 2015-09-16 마이오실린(myoc) 녹내장을 치료하기 위한 아데노-관련 바이러스 벡터

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201462051299P 2014-09-16 2014-09-16
US62/051,299 2014-09-16
PCT/US2015/050515 WO2016044478A1 (en) 2014-09-16 2015-09-16 Adeno-associated viral vectors for treating myocilin (myoc) glaucoma

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KR1020237042363A Division KR20230172610A (ko) 2014-09-16 2015-09-16 마이오실린(myoc) 녹내장을 치료하기 위한 아데노-관련 바이러스 벡터

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KR20170054493A KR20170054493A (ko) 2017-05-17
KR102612871B1 true KR102612871B1 (ko) 2023-12-13

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KR1020237042363A Pending KR20230172610A (ko) 2014-09-16 2015-09-16 마이오실린(myoc) 녹내장을 치료하기 위한 아데노-관련 바이러스 벡터
KR1020177010012A Active KR102612871B1 (ko) 2014-09-16 2015-09-16 마이오실린(myoc) 녹내장을 치료하기 위한 아데노-관련 바이러스 벡터

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US (1) US10821193B2 (enExample)
EP (1) EP3194601B1 (enExample)
JP (2) JP6673923B2 (enExample)
KR (2) KR20230172610A (enExample)
CN (2) CN116585492A (enExample)
AU (2) AU2015317756B2 (enExample)
BR (1) BR112017005235B1 (enExample)
IL (1) IL251178B (enExample)
MX (2) MX2017003426A (enExample)
RU (2) RU2746991C2 (enExample)
SG (3) SG10201902285SA (enExample)
ZA (1) ZA201701881B (enExample)

Families Citing this family (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
SG10201902285SA (en) 2014-09-16 2019-04-29 Genzyme Corp Adeno-associated viral vectors for treating myocilin (myoc) glaucoma
US11845952B2 (en) * 2018-02-07 2023-12-19 Nippon Medical School Foundation Adeno-associated virus vector
US20210030719A1 (en) * 2018-02-22 2021-02-04 University Of South Florida Grp94 inhibitors to treat steroid-induced ocular hypertensions and glaucomas
AU2020229871B2 (en) 2019-02-28 2023-05-18 Regeneron Pharmaceuticals, Inc. Adeno-associated virus vectors for the delivery of therapeutics
EP4255457A4 (en) * 2020-12-03 2025-05-21 University of Massachusetts DEVELOPMENT OF NEW GENE THERAPEUTIC AGENTS FOR FIBRODYSPLASIA OSSIFICANS PROGRESSIVA
CN113032010B (zh) * 2021-03-12 2022-09-20 歌尔科技有限公司 命令的传输控制方法、终端及计算机可读存储介质
CA3224868A1 (en) * 2021-06-28 2023-01-05 Alnylam Pharmaceuticals, Inc. Compositions and methods for silencing myoc expression
WO2023114446A2 (en) * 2021-12-16 2023-06-22 Peter Koulen A machine learning-based framework using electroretinography for detecting early-stage glaucoma
CN117343152B (zh) * 2023-04-18 2024-06-04 上海本导基因技术有限公司 一种用于治疗青光眼疾病的慢病毒样颗粒
CN118546217B (zh) * 2024-07-25 2024-10-29 广州译码基因科技有限公司 靶向小梁网、角膜缘及rpe的aav衣壳蛋白及其应用

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2008075796A1 (ja) 2006-12-21 2008-06-26 Kyowa Hakko Kirin Co., Ltd. 血球回復促進剤
JP2010540564A (ja) 2007-10-01 2010-12-24 アルコン リサーチ， リミテッド 自己相補的なａａｖが媒介する眼の障害を処置または予防するための干渉ｒｎａ分子の送達
JP2014518614A (ja) 2011-04-22 2014-08-07 ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア 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

Family Cites Families (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP1434796A2 (en) 2001-10-03 2004-07-07 Incyte Genomics, Inc. Secreted proteins
GB0403600D0 (en) * 2004-02-18 2004-03-24 Trinity College Dublin Methods and reagents for treating disease
WO2005117938A2 (en) * 2004-04-13 2005-12-15 Regents Of The University Of Minnesota Methods of treating ocular conditions
GB201106395D0 (en) * 2011-04-14 2011-06-01 Hubrecht Inst Compounds
EP2554662A1 (en) * 2011-08-05 2013-02-06 M Maria Pia Cosma Methods of treatment of retinal degeneration diseases
WO2013120056A1 (en) * 2012-02-11 2013-08-15 Genentech, Inc. R-spondin translocations and methods using the same
JP6385920B2 (ja) * 2012-05-09 2018-09-05 オレゴン ヘルス アンド サイエンス ユニバーシティー アデノ随伴ウイルスプラスミド及びベクター
SG10201902285SA (en) 2014-09-16 2019-04-29 Genzyme Corp Adeno-associated viral vectors for treating myocilin (myoc) glaucoma

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2008075796A1 (ja) 2006-12-21 2008-06-26 Kyowa Hakko Kirin Co., Ltd. 血球回復促進剤
JP2010540564A (ja) 2007-10-01 2010-12-24 アルコン リサーチ， リミテッド 自己相補的なａａｖが媒介する眼の障害を処置または予防するための干渉ｒｎａ分子の送達
JP2014518614A (ja) 2011-04-22 2014-08-07 ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア 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

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