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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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 (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=)

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
SG10201800541SA (en) 2010-04-23 2018-03-28 Univ Florida Raav-guanylate cyclase compositions and methods for treating leber's congenital amaurosis-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
PL4012035T3 (pl) * 2014-09-16 2025-03-24 Genzyme Corporation Wektory wirusowe związane z adenowirusami do leczenia jaskry miocylinowej (myoc)
EP4012035B1 (en) * 2014-09-16 2024-11-06 Genzyme Corporation Adeno-associated viral vectors for treating myocilin (myoc) glaucoma
KR20220076510A (ko) 2019-10-08 2022-06-08 트러스티스 오브 보스톤 칼리지 다수의 서로 다른 비천연 아미노산을 함유하는 단백질 및 이러한 단백질의 제조 및 사용 방법
CN115484978A (zh) 2020-03-05 2022-12-16 尼奥克斯医疗有限公司 使用免疫细胞治疗癌症的方法和组合物
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

Family Cites Families (20)

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Publication number Priority date Publication date Assignee Title
ATE318923T1 (de) * 2000-06-01 2006-03-15 Univ North Carolina Doppelsträngige parvovirus-vektoren
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)
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)
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
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
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
TW200639252A (en) * 2005-02-01 2006-11-16 Alcon Inc RNAi-mediated inhibition of ocular hypertension targets
WO2006099353A1 (en) * 2005-03-11 2006-09-21 Alcon, Inc. Rnai-mediated inhibition of frizzled related protein-1 for treatment of 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
TW201336514A (zh) * 2006-04-13 2013-09-16 Alcon Res Ltd ＲＮＡ干擾（ＲＮＡｉ）所媒介之與脾酪胺酸激酶相關之發炎症狀的抑制作用（二）
WO2007127428A2 (en) * 2006-04-28 2007-11-08 University Of Florida Research Foundation, Inc. Double-stranded/self-complementary vectors with a truncated cba promoter and methods of gene delivery
US7732421B2 (en) * 2006-05-19 2010-06-08 Alcon Research, Ltd. RNAI-mediated inhibition of tumor necrosis factor α-related conditions
KR20090042297A (ko) * 2006-08-24 2009-04-29 알콘 리서치, 리미티드 ＩＯＰ―관련 증상을 치료하기 위한 그렘린의 ＲＮＡｉ 매개 억제
US7872119B2 (en) * 2007-02-26 2011-01-18 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
KR20100061792A (ko) 2010-06-09
CA2694091A1 (en) 2009-04-09
US20090087413A1 (en) 2009-04-02
AU2008308784A1 (en) 2009-04-09
ZA201000423B (en) 2011-03-30
BRPI0817937A2 (pt) 2015-04-07
RU2010117178A (ru) 2011-11-10
EP2192926A1 (en) 2010-06-09
AU2008308784B2 (en) 2013-07-18
JP2010540564A (ja) 2010-12-24
WO2009046059A1 (en) 2009-04-09
MX2010001608A (es) 2010-03-15

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Application publication date: 20100825