RU2746991C2 - Аденоассоциированные вирусные векторы для лечения миоцилиновой (myoc) глаукомы - Google Patents

Аденоассоциированные вирусные векторы для лечения миоцилиновой (myoc) глаукомы Download PDF

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RU2746991C2
RU2746991C2 RU2020111038A RU2020111038A RU2746991C2 RU 2746991 C2 RU2746991 C2 RU 2746991C2 RU 2020111038 A RU2020111038 A RU 2020111038A RU 2020111038 A RU2020111038 A RU 2020111038A RU 2746991 C2 RU2746991 C2 RU 2746991C2
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myoc
raav
aav2
aav
nucleic acid
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RU2020111038A
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RU2020111038A3 (OSRAM
RU2020111038A (ru
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Питер ПЕЧАН
Абрахам СКАРИА
Джеффри АРДИНГЕР
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Джензим Корпорейшн
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    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
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RU2020111038A 2014-09-16 2015-09-16 Аденоассоциированные вирусные векторы для лечения миоцилиновой (myoc) глаукомы RU2746991C2 (ru)

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US201462051299P 2014-09-16 2014-09-16
US62/051,299 2014-09-16

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RU2020111038A RU2020111038A (ru) 2020-04-29
RU2020111038A3 RU2020111038A3 (OSRAM) 2020-10-15
RU2746991C2 true RU2746991C2 (ru) 2021-04-23

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RU2017112972A RU2718047C2 (ru) 2014-09-16 2015-09-16 Аденоассоциированные вирусные векторы для лечения миоцилиновой (myoc) глаукомы

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US (1) US10821193B2 (OSRAM)
EP (1) EP3194601B1 (OSRAM)
JP (2) JP6673923B2 (OSRAM)
KR (2) KR102612871B1 (OSRAM)
CN (2) CN107001436B (OSRAM)
AU (2) AU2015317756B2 (OSRAM)
BR (1) BR112017005235B1 (OSRAM)
IL (1) IL251178B (OSRAM)
MX (2) MX2017003426A (OSRAM)
RU (2) RU2746991C2 (OSRAM)
SG (3) SG10201913430VA (OSRAM)
ZA (1) ZA201701881B (OSRAM)

Families Citing this family (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR102612871B1 (ko) 2014-09-16 2023-12-13 젠자임 코포레이션 마이오실린(myoc) 녹내장을 치료하기 위한 아데노-관련 바이러스 벡터
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
JP2022522756A (ja) 2019-02-28 2022-04-20 リジェネロン・ファーマシューティカルズ・インコーポレイテッド 治療薬の送達のためのアデノ随伴ウイルスベクター
WO2022120080A1 (en) * 2020-12-03 2022-06-09 University Of Massachusetts Development of novel gene therapeutics 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
US20240318175A1 (en) * 2021-06-28 2024-09-26 Alnylam Pharmaceuticals, Inc. Compositions and Methods for Silencing MYOC Expression
US20250087355A1 (en) * 2021-12-16 2025-03-13 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
WO2005079815A2 (en) * 2004-02-18 2005-09-01 The Provost, Fellows And Scholars Of The College Of The Holy And Undivided Trinity Of Queen Elizabeth Near Dublin Methods and reagents for treating autosomal dominant diseases of the eye
WO2005117938A2 (en) * 2004-04-13 2005-12-15 Regents Of The University Of Minnesota Methods of treating ocular conditions
WO2009046059A1 (en) * 2007-10-01 2009-04-09 Alcon Research, Ltd. Self complementary aav-mediated delivery of interfering rna molecules to treat or prevent ocular disorders

Family Cites Families (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2005514917A (ja) 2001-10-03 2005-05-26 インサイト・ゲノミックス・インコーポレイテッド 分泌タンパク質
WO2008075796A1 (ja) 2006-12-21 2008-06-26 Kyowa Hakko Kirin Co., Ltd. 血球回復促進剤
GB201106395D0 (en) * 2011-04-14 2011-06-01 Hubrecht Inst Compounds
RS66184B1 (sr) 2011-04-22 2024-12-31 Univ California Adeno-povezani virioni virusa sa varijantama kapsida i postupci za njihovu primenu
EP2554662A1 (en) * 2011-08-05 2013-02-06 M Maria Pia Cosma Methods of treatment of retinal degeneration diseases
EP2812350B1 (en) * 2012-02-11 2019-04-03 F.Hoffmann-La Roche Ag R-spondin translocations and methods using the same
EP2847337A4 (en) * 2012-05-09 2016-04-27 Univ Oregon Health & Science ADENO ASSOCIATED VIRUS PLASMIDS AND VECTORS
KR102612871B1 (ko) 2014-09-16 2023-12-13 젠자임 코포레이션 마이오실린(myoc) 녹내장을 치료하기 위한 아데노-관련 바이러스 벡터

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2005079815A2 (en) * 2004-02-18 2005-09-01 The Provost, Fellows And Scholars Of The College Of The Holy And Undivided Trinity Of Queen Elizabeth Near Dublin Methods and reagents for treating autosomal dominant diseases of the eye
WO2005117938A2 (en) * 2004-04-13 2005-12-15 Regents Of The University Of Minnesota Methods of treating ocular conditions
WO2009046059A1 (en) * 2007-10-01 2009-04-09 Alcon Research, Ltd. Self complementary aav-mediated delivery of interfering rna molecules to treat or prevent ocular disorders

Non-Patent Citations (3)

* Cited by examiner, † Cited by third party
Title
KWON HS et al. Myocilin is a modulator of Wnt signaling. Mol Cell Biol., 2009, 29(8), p.2139-54. *
LOCHRIE MA et al. Mutations on the external surfaces of adeno-associated virus type 2 capsids that affect transduction and neutralization. J Virol., 2006, 80(2), p.821-834. *
LOCHRIE MA et al. Mutations on the external surfaces of adeno-associated virus type 2 capsids that affect transduction and neutralization. J Virol., 2006, 80(2), p.821-834. KWON HS et al. Myocilin is a modulator of Wnt signaling. Mol Cell Biol., 2009, 29(8), p.2139-54. *

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JP2017529395A (ja) 2017-10-05
SG10201913430VA (en) 2020-03-30
RU2020111038A3 (OSRAM) 2020-10-15
MX2017003426A (es) 2017-07-28
AU2015317756B2 (en) 2022-01-13
US20170304465A1 (en) 2017-10-26
KR20170054493A (ko) 2017-05-17
ZA201701881B (en) 2023-09-27
AU2015317756A1 (en) 2017-04-13
CN107001436A (zh) 2017-08-01
BR112017005235A2 (pt) 2017-12-12
CN107001436B (zh) 2023-01-06
AU2022202413A1 (en) 2022-05-05
SG10201902285SA (en) 2019-04-29
US10821193B2 (en) 2020-11-03
MX2023005998A (es) 2023-06-08
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