ZA201000423B - Self complementary aav-mediated delivery of interfering rna molecules to treator prevent ocular disorders - Google Patents

Self complementary aav-mediated delivery of interfering rna molecules to treator prevent ocular disorders

Info

Publication number
ZA201000423B
ZA201000423B ZA2010/00423A ZA201000423A ZA201000423B ZA 201000423 B ZA201000423 B ZA 201000423B ZA 2010/00423 A ZA2010/00423 A ZA 2010/00423A ZA 201000423 A ZA201000423 A ZA 201000423A ZA 201000423 B ZA201000423 B ZA 201000423B
Authority
ZA
South Africa
Prior art keywords
rna molecules
interfering rna
mediated delivery
ocular disorders
self complementary
Prior art date
Application number
ZA2010/00423A
Other languages
English (en)
Inventor
Allan R Shepard
Original Assignee
Alcon Res Ltd
Priority date (The priority date 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 date listed.)
Filing date
Publication date
Application filed by Alcon Res Ltd filed Critical Alcon Res Ltd
Publication of ZA201000423B publication Critical patent/ZA201000423B/en

Links

Classifications

    • 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

Landscapes

  • 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)
ZA2010/00423A 2007-10-01 2010-01-20 Self complementary aav-mediated delivery of interfering rna molecules to treator prevent ocular disorders ZA201000423B (en)

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 (1)

Publication Number Publication Date
ZA201000423B true ZA201000423B (en) 2011-03-30

Family

ID=40042637

Family Applications (1)

Application Number Title Priority Date Filing Date
ZA2010/00423A ZA201000423B (en) 2007-10-01 2010-01-20 Self complementary aav-mediated delivery of interfering rna molecules to treator prevent ocular disorders

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
HUE041571T2 (hu) 2010-04-23 2019-05-28 Univ Florida RAAV-guanilát-cikláz-készítmények és ejárások 1-es típusú Leber-féle veleszületett vakság (LCA1) kezelésére
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)
IL251178B (en) * 2014-09-16 2022-07-01 Genzyme Corp Adeno-associated viral vector for the treatment of glaucoma myocilin (myoc)
GB201809588D0 (en) * 2018-06-12 2018-07-25 Univ Bristol Materials and methods for modulating intraocular and intracranial pressure
US12680122B2 (en) 2019-10-08 2026-07-14 Trustees Of Boston College Proteins containing multiple, different unnatural amino acids and methods of making and using such proteins
JP2023517011A (ja) 2020-03-05 2023-04-21 ネオティーエックス セラピューティクス リミテッド 免疫細胞を用いて癌を治療するための方法および組成物
US20230235326A1 (en) * 2020-06-05 2023-07-27 INSERM (Institut National de la Santé et de la Recherche Médicale) 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
US7465583B2 (en) * 2000-06-01 2008-12-16 The University Of North Carolina At Chapel Hill Duplexed parvovirus vectors
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)
SG145713A1 (en) * 2003-08-13 2008-09-29 Univ Illinois SILENCING OF TGFss TYPE II RECEPTOR EXPRESSION BY SIRNA
WO2006042252A2 (en) * 2004-10-08 2006-04-20 Potentia Pharmeceuticals, 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
CA2598234A1 (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
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
RU2469090C2 (ru) * 2006-05-19 2012-12-10 Алькон Рисерч, Лтд. iPHK-ОПОСРЕДОВАННОЕ ИНГИБИРОВАНИЕ СВЯЗАННЫХ С ФАКТОРОМ НЕКРОЗА ОПУХОЛИ АЛЬФА СОСТОЯНИЙ
US20080051361A1 (en) * 2006-08-24 2008-02-28 Alcon Manufacturing, Ltd. Rnai-mediated inhibition of gremlin for treatment of iop-related conditions
WO2008106102A2 (en) * 2007-02-26 2008-09-04 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
AU2008308784A1 (en) 2009-04-09
CA2694091A1 (en) 2009-04-09
MX2010001608A (es) 2010-03-15
AU2008308784B2 (en) 2013-07-18
KR20100061792A (ko) 2010-06-09
WO2009046059A1 (en) 2009-04-09
BRPI0817937A2 (pt) 2015-04-07
RU2010117178A (ru) 2011-11-10
US20090087413A1 (en) 2009-04-02
JP2010540564A (ja) 2010-12-24
EP2192926A1 (en) 2010-06-09
CN101815536A (zh) 2010-08-25

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