JP2010540564A - 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 - Google Patents

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 Download PDF

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JP2010540564A
JP2010540564A JP2010527251A JP2010527251A JP2010540564A JP 2010540564 A JP2010540564 A JP 2010540564A JP 2010527251 A JP2010527251 A JP 2010527251A JP 2010527251 A JP2010527251 A JP 2010527251A JP 2010540564 A JP2010540564 A JP 2010540564A
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interfering rna
eye
scaav
vector
ocular
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アラン アール. シェパード,
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アルコン リサーチ, リミテッド
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    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00Drugs for disorders of the senses
    • A61P27/02Ophthalmic agents
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00Drugs for disorders of the senses
    • A61P27/02Ophthalmic agents
    • A61P27/04Artificial tears; Irrigation solutions
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00Drugs for disorders of the senses
    • A61P27/02Ophthalmic agents
    • A61P27/06Antiglaucoma agents or miotics
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00Drugs for disorders of the senses
    • A61P27/02Ophthalmic agents
    • A61P27/14Decongestants or antiallergics
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector

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  • Health & Medical Sciences (AREA)
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  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Ophthalmology & Optometry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • General Engineering & Computer Science (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Zoology (AREA)
  • Biomedical Technology (AREA)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • Virology (AREA)
  • Molecular Biology (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Biochemistry (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
JP2010527251A 2007-10-01 2008-10-01 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 Pending JP2010540564A (ja)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US97655207P 2007-10-01 2007-10-01
PCT/US2008/078380 WO2009046059A1 (en) 2007-10-01 2008-10-01 Self complementary aav-mediated delivery of interfering rna molecules to treat or prevent ocular disorders

Publications (2)

Publication Number Publication Date
JP2010540564A true JP2010540564A (ja) 2010-12-24
JP2010540564A5 JP2010540564A5 (https=) 2012-09-13

Family

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Family Applications (1)

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JP2010527251A Pending JP2010540564A (ja) 2007-10-01 2008-10-01 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

Country Status (12)

Country Link
US (1) US20090087413A1 (https=)
EP (1) EP2192926A1 (https=)
JP (1) JP2010540564A (https=)
KR (1) KR20100061792A (https=)
CN (1) CN101815536A (https=)
AU (1) AU2008308784B2 (https=)
BR (1) BRPI0817937A2 (https=)
CA (1) CA2694091A1 (https=)
MX (1) MX2010001608A (https=)
RU (1) RU2010117178A (https=)
WO (1) WO2009046059A1 (https=)
ZA (1) ZA201000423B (https=)

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KR20170054493A (ko) * 2014-09-16 2017-05-17 젠자임 코포레이션 마이오실린(myoc) 녹내장을 치료하기 위한 아데노-관련 바이러스 벡터
JP2021526839A (ja) * 2018-06-12 2021-10-11 ザ ユニバーシティ オブ ブリストル 眼圧及び頭蓋内圧を調節するための材料及び方法

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EP2019143A1 (en) 2007-07-23 2009-01-28 Genethon CNS gene delivery using peripheral administration of AAV vectors
EP2058401A1 (en) 2007-10-05 2009-05-13 Genethon Widespread gene delivery to motor neurons using peripheral injection of AAV vectors
EP2287323A1 (en) 2009-07-31 2011-02-23 Association Institut de Myologie Widespread gene delivery to the retina using systemic administration of AAV vectors
HRP20190144T1 (hr) * 2010-04-23 2019-03-22 University Of Florida Research Foundation, Inc. Pripravci raav-gvanilat ciklaze i postupci za liječenje leberove urođene amauroze-1 (lca1)
CN103505743A (zh) 2012-06-21 2014-01-15 北京命码生科科技有限公司 含功能性microRNA/siRNA的细胞微粒子及其应用
EP4012035B1 (en) * 2014-09-16 2024-11-06 Genzyme Corporation Adeno-associated viral vectors for treating myocilin (myoc) glaucoma
PT4012035T (pt) * 2014-09-16 2025-02-11 Genzyme Corp Vetores virais adenoassociados para tratar o glaucoma de miocilina (mioc)
AU2020361533A1 (en) 2019-10-08 2022-04-28 Trustees Of Boston College Proteins containing multiple, different unnatural amino acids and methods of making and using such proteins
IL296103A (en) 2020-03-05 2022-11-01 Neotx Therapeutics Ltd Methods and compositions for treating cancer with immune cells
WO2021245224A1 (en) * 2020-06-05 2021-12-09 INSERM (Institut National de la Santé et de la Recherche Médicale) Methods and pharmaceutical compositions for treating ocular diseases

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US20040209832A1 (en) * 2001-11-30 2004-10-21 Mcswiggen James RNA interference mediated inhibition of vascular endothelial growth factor and vascular endothelial growth factor receptor gene expression using short interfering nucleic acid (siNA)
US20050048529A1 (en) * 2002-02-20 2005-03-03 Sirna Therapeutics, Inc. RNA interference mediated inhibition of intercellular adhesion molecule (ICAM) gene expression using short interfering nucleic acid (siNA)
US20040198682A1 (en) * 2001-11-30 2004-10-07 Mcswiggen James RNA interference mediated inhibition of placental growth factor gene expression using short interfering nucleic acid (siNA)
US20060229266A1 (en) * 2003-08-13 2006-10-12 Kumar Nalin M Silencing of tgf-beta receptor type II expression by sirna
WO2006042252A2 (en) * 2004-10-08 2006-04-20 Potentia Pharmeceuticals, Inc. Viral complement control proteins for eye disorders
TWI401316B (zh) * 2004-12-23 2013-07-11 Alcon Inc 用於治療青光眼之血清澱粉樣蛋白A的RNAi抑制作用
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JPN6013023944; 'Mechanisms of AAV transduction in glaucoma-associated human trabecular meshwork cells.' J Gene Med. 8(5), 2006, 589-602 *
JPN6013023945; 'AAV hybrid serotypes: improved vectors for gene delivery.' Curr Gene Ther. 5(3), 2005, 299-310 *
JPN6013023948; 'Inhibition of Ocular Angiogenesis by siRNA Targeting Vascular Endothelial Growth Factor Pathway Gene' Am J Pathol. 165(6), 2004, 2177-2185 *
JPN6013023949; 'Potential applications for RNAi to probe pathogenesis and develop new treatments for ocular disorder' Gene Therapy 13(6), 2006, 559-562 *

Cited By (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR20170054493A (ko) * 2014-09-16 2017-05-17 젠자임 코포레이션 마이오실린(myoc) 녹내장을 치료하기 위한 아데노-관련 바이러스 벡터
JP2017529395A (ja) * 2014-09-16 2017-10-05 ジェンザイム・コーポレーション ミオシリン(myoc)緑内障を処置するためのアデノ随伴ウイルスベクター
KR102612871B1 (ko) 2014-09-16 2023-12-13 젠자임 코포레이션 마이오실린(myoc) 녹내장을 치료하기 위한 아데노-관련 바이러스 벡터
KR20230172610A (ko) * 2014-09-16 2023-12-22 젠자임 코포레이션 마이오실린(myoc) 녹내장을 치료하기 위한 아데노-관련 바이러스 벡터
KR102916865B1 (ko) 2014-09-16 2026-01-23 젠자임 코포레이션 마이오실린(myoc) 녹내장을 치료하기 위한 아데노-관련 바이러스 벡터
JP2021526839A (ja) * 2018-06-12 2021-10-11 ザ ユニバーシティ オブ ブリストル 眼圧及び頭蓋内圧を調節するための材料及び方法
JP7430917B2 (ja) 2018-06-12 2024-02-14 ザ ユニバーシティ オブ ブリストル 眼圧及び頭蓋内圧を調節するための材料及び方法

Also Published As

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MX2010001608A (es) 2010-03-15
US20090087413A1 (en) 2009-04-02
BRPI0817937A2 (pt) 2015-04-07
CA2694091A1 (en) 2009-04-09
RU2010117178A (ru) 2011-11-10
CN101815536A (zh) 2010-08-25
KR20100061792A (ko) 2010-06-09
ZA201000423B (en) 2011-03-30
WO2009046059A1 (en) 2009-04-09
AU2008308784A1 (en) 2009-04-09
AU2008308784B2 (en) 2013-07-18
EP2192926A1 (en) 2010-06-09

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