CN101815536A - 自补aav介导的干扰rna分子递送以治疗或预防眼病 - Google Patents

自补aav介导的干扰rna分子递送以治疗或预防眼病 Download PDF

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Publication number
CN101815536A
CN101815536A CN200880110028A CN200880110028A CN101815536A CN 101815536 A CN101815536 A CN 101815536A CN 200880110028 A CN200880110028 A CN 200880110028A CN 200880110028 A CN200880110028 A CN 200880110028A CN 101815536 A CN101815536 A CN 101815536A
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CN
China
Prior art keywords
eye
scaav
rna
sirna
interfering rna
Prior art date
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Pending
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CN200880110028A
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English (en)
Chinese (zh)
Inventor
A·R·谢帕德
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Alcon Research LLC
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Alcon Research LLC
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Application filed by Alcon Research LLC filed Critical Alcon Research LLC
Publication of CN101815536A publication Critical patent/CN101815536A/zh
Pending legal-status Critical Current

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    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/02—Ophthalmic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/02—Ophthalmic agents
    • A61P27/04—Artificial tears; Irrigation solutions
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/02—Ophthalmic agents
    • A61P27/06—Antiglaucoma agents or miotics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/02—Ophthalmic agents
    • A61P27/14—Decongestants or antiallergics
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use 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)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Chemical & Material Sciences (AREA)
  • 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)
CN200880110028A 2007-10-01 2008-10-01 自补aav介导的干扰rna分子递送以治疗或预防眼病 Pending CN101815536A (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US97655207P 2007-10-01 2007-10-01
US60/976,552 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 (1)

Publication Number Publication Date
CN101815536A true CN101815536A (zh) 2010-08-25

Family

ID=40042637

Family Applications (1)

Application Number Title Priority Date Filing Date
CN200880110028A Pending CN101815536A (zh) 2007-10-01 2008-10-01 自补aav介导的干扰rna分子递送以治疗或预防眼病

Country Status (12)

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

Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN107001436A (zh) * 2014-09-16 2017-08-01 建新公司 用于治疗肌纤蛋白(myoc)青光眼的腺伴随病毒载体
CN112424368A (zh) * 2018-06-12 2021-02-26 布利斯脱大学 用于调节眼内压和颅内压的材料和方法

Families Citing this family (10)

* Cited by examiner, † Cited by third party
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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
EA202091105A1 (ru) * 2010-04-23 2020-12-30 Юниверсити Оф Флорида Рисерч Фаундейшн, Инк. Композиции гуанилатциклазы и способы лечения врожденного амавроза лебера типа 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
CA2961523A1 (en) * 2014-09-16 2016-03-24 Genzyme Corporation Adeno-associated viral vectors for treating myocilin (myoc) glaucoma
US20220403438A1 (en) 2019-10-08 2022-12-22 Trustees Of Boston College Proteins containing multiple, different unnatural amino acids and methods of making and using such proteins
MX2022010936A (es) 2020-03-05 2022-11-16 Neotx Therapeutics Ltd ³métodos y composiciones para el tratamiento del cáncer con células inmunológicas.
EP4161583A1 (en) * 2020-06-05 2023-04-12 Institut National de la Santé et de la Recherche Médicale (INSERM) Methods and pharmaceutical compositions for treating ocular diseases

Family Cites Families (20)

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Publication number Priority date Publication date Assignee Title
ES2256265T3 (es) * 2000-06-01 2006-07-16 University Of North Carolina At Chapel Hill Vectores de parvovirus duplicados.
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)
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)
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)
WO2005019422A2 (en) * 2003-08-13 2005-03-03 The Board Of Trustees Of The University Of Illinois Silencing of tgf-beta receptor type ii expression by sirna
US7947267B2 (en) * 2004-10-08 2011-05-24 Potentia Pharmaceuticals, Inc. Viral complement control proteins for eye disorders
TWI386225B (zh) * 2004-12-23 2013-02-21 Alcon Inc 用於治療眼睛病症的結締組織生長因子（ＣＴＧＦ）ＲＮＡ干擾（ＲＮＡｉ）抑制技術
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
TW200639253A (en) * 2005-02-01 2006-11-16 Alcon Inc RNAi-mediated inhibition of ocular targets
BRPI0609206A2 (pt) * 2005-03-11 2010-03-02 Alcon Inc inibiÇço mediada por rnai de proteÍna relacionada frizzled-1 para tratamento de glaucoma
GB0521351D0 (en) * 2005-10-20 2005-11-30 Genomica Sau Modulation of TRPV expression levels
AR057252A1 (es) * 2005-12-27 2007-11-21 Alcon Mfg Ltd Inhibicion de rho quinasa mediada por arni para el tratamiento de trastornos oculares
TW200731980A (en) * 2005-12-29 2007-09-01 Alcon Mfg Ltd RNAi-mediated inhibition of HIF1A for treatment of ocular angiogenesis
TW200808360A (en) * 2006-04-13 2008-02-16 Alcon Mfg Ltd RNAi-mediated inhibition of spleen tyrosine kinase-related inflammatory conditions
US8298818B2 (en) * 2006-04-28 2012-10-30 University Of Florida Research Foundation, Inc. Self-complementary adeno-associated virus having a truncated CMV-chicken β-actin promoter
AU2007253776B2 (en) * 2006-05-19 2012-04-26 Arrowhead Research Corporation RNAi-mediated inhibition of tumor necrosis factor alpha-related conditions
BRPI0715821A2 (pt) * 2006-08-24 2013-07-23 Alcon Res Ltd inibiÇço de gremlina mediada por rnai para tratamento de condiÇÕes relacionadas À iop
EP2137205A2 (en) * 2007-02-26 2009-12-30 Quark Pharmaceuticals, Inc. Inhibitors of rtp801 and their use in disease treatment
US7973019B1 (en) * 2007-10-03 2011-07-05 Alcon Research, Ltd. Transferrin/transferrin receptor-mediated siRNA delivery
TW200932274A (en) * 2007-12-18 2009-08-01 Alcon Res Ltd Interfering RNA delivery system and uses thereof

Cited By (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN107001436A (zh) * 2014-09-16 2017-08-01 建新公司 用于治疗肌纤蛋白(myoc)青光眼的腺伴随病毒载体
CN107001436B (zh) * 2014-09-16 2023-01-06 建新公司 用于治疗肌纤蛋白(myoc)青光眼的腺伴随病毒载体
CN112424368A (zh) * 2018-06-12 2021-02-26 布利斯脱大学 用于调节眼内压和颅内压的材料和方法

Also Published As

Publication number Publication date
CA2694091A1 (en) 2009-04-09
AU2008308784B2 (en) 2013-07-18
ZA201000423B (en) 2011-03-30
JP2010540564A (ja) 2010-12-24
BRPI0817937A2 (pt) 2015-04-07
MX2010001608A (es) 2010-03-15
US20090087413A1 (en) 2009-04-02
WO2009046059A1 (en) 2009-04-09
KR20100061792A (ko) 2010-06-09
RU2010117178A (ru) 2011-11-10
AU2008308784A1 (en) 2009-04-09
EP2192926A1 (en) 2010-06-09

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Application publication date: 20100825