KR20100061792A - 안 질환을 치료 또는 예방하기 위한 간섭 rna 분자의 자기-상보적 aav 매개 전달 - Google Patents

안 질환을 치료 또는 예방하기 위한 간섭 rna 분자의 자기-상보적 aav 매개 전달 Download PDF

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Publication number
KR20100061792A
KR20100061792A KR1020107003620A KR20107003620A KR20100061792A KR 20100061792 A KR20100061792 A KR 20100061792A KR 1020107003620 A KR1020107003620 A KR 1020107003620A KR 20107003620 A KR20107003620 A KR 20107003620A KR 20100061792 A KR20100061792 A KR 20100061792A
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KR
South Korea
Prior art keywords
interfering rna
scaav
ocular
eye
sirna
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Withdrawn
Application number
KR1020107003620A
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English (en)
Korean (ko)
Inventor
앨런 알. 쉐퍼드
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알콘 리서치, 리미티드
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Application filed by 알콘 리서치, 리미티드 filed Critical 알콘 리서치, 리미티드
Publication of KR20100061792A publication Critical patent/KR20100061792A/ko
Withdrawn legal-status Critical Current

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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)
  • 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)
KR1020107003620A 2007-10-01 2008-10-01 안 질환을 치료 또는 예방하기 위한 간섭 rna 분자의 자기-상보적 aav 매개 전달 Withdrawn KR20100061792A (ko)

Applications Claiming Priority (2)

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

Publications (1)

Publication Number Publication Date
KR20100061792A true KR20100061792A (ko) 2010-06-09

Family

ID=40042637

Family Applications (1)

Application Number Title Priority Date Filing Date
KR1020107003620A Withdrawn KR20100061792A (ko) 2007-10-01 2008-10-01 안 질환을 치료 또는 예방하기 위한 간섭 rna 분자의 자기-상보적 aav 매개 전달

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)

Families Citing this family (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
KR102612871B1 (ko) 2014-09-16 2023-12-13 젠자임 코포레이션 마이오실린(myoc) 녹내장을 치료하기 위한 아데노-관련 바이러스 벡터
CA2961523A1 (en) * 2014-09-16 2016-03-24 Genzyme Corporation Adeno-associated viral vectors for treating myocilin (myoc) glaucoma
GB201809588D0 (en) * 2018-06-12 2018-07-25 Univ Bristol Materials and methods for modulating intraocular and intracranial pressure
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)

* Cited by examiner, † Cited by third party
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

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
RU2010117178A (ru) 2011-11-10
AU2008308784A1 (en) 2009-04-09
CN101815536A (zh) 2010-08-25
EP2192926A1 (en) 2010-06-09

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Legal Events

Date Code Title Description
PA0105 International application

Patent event date: 20100218

Patent event code: PA01051R01D

Comment text: International Patent Application

PG1501 Laying open of application
PC1203 Withdrawal of no request for examination
WITN Application deemed withdrawn, e.g. because no request for examination was filed or no examination fee was paid