HK1072074A1 - Means and methods for the specific modulation of target genes in the eye - Google Patents

Means and methods for the specific modulation of target genes in the eye

Info

Publication number
HK1072074A1
HK1072074A1 HK05104837.4A HK05104837A HK1072074A1 HK 1072074 A1 HK1072074 A1 HK 1072074A1 HK 05104837 A HK05104837 A HK 05104837A HK 1072074 A1 HK1072074 A1 HK 1072074A1
Authority
HK
Hong Kong
Prior art keywords
eye
methods
target genes
specific modulation
modulation
Prior art date
Application number
HK05104837.4A
Other languages
English (en)
Inventor
Karina Drumm
Stefan Hubert Schloer
Frank Goehring
Original Assignee
Acuity Pharmaceuticals Inc
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 Acuity Pharmaceuticals Inc filed Critical Acuity Pharmaceuticals Inc
Publication of HK1072074A1 publication Critical patent/HK1072074A1/xx

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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C12N15/1136—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing against growth factors, growth regulators, cytokines, lymphokines or hormones
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/1703—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • A61K38/1709—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/04—Centrally acting analgesics, e.g. opioids
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/06—Antimigraine agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • A61P25/16—Anti-Parkinson drugs
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/20—Hypnotics; Sedatives
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/22—Anxiolytics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/24—Antidepressants
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/30—Drugs for disorders of the nervous system for treating abuse or dependence
    • A61P25/32—Alcohol-abuse
    • 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
    • A61P37/00—Drugs for immunological or allergic disorders
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A61P9/10—Drugs for disorders of the cardiovascular system for treating ischaemic or atherosclerotic diseases, e.g. antianginal drugs, coronary vasodilators, drugs for myocardial infarction, retinopathy, cerebrovascula insufficiency, renal arteriosclerosis
    • 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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/111—General methods applicable to biologically active non-coding nucleic acids
    • 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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N33/00—Investigating or analysing materials by specific methods not covered by groups G01N1/00 - G01N31/00
    • G01N33/48—Biological material, e.g. blood, urine; Haemocytometers
    • G01N33/50—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
    • G01N33/5005—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving human or animal cells
    • G01N33/5091—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving human or animal cells for testing the pathological state of an organism
    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N33/00—Investigating or analysing materials by specific methods not covered by groups G01N1/00 - G01N31/00
    • G01N33/48—Biological material, e.g. blood, urine; Haemocytometers
    • G01N33/50—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
    • G01N33/68—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving proteins, peptides or amino acids
    • G01N33/6893—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving proteins, peptides or amino acids related to diseases not provided for elsewhere
    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N33/00—Investigating or analysing materials by specific methods not covered by groups G01N1/00 - G01N31/00
    • G01N33/48—Biological material, e.g. blood, urine; Haemocytometers
    • G01N33/50—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
    • G01N33/68—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving proteins, peptides or amino acids
    • G01N33/6893—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving proteins, peptides or amino acids related to diseases not provided for elsewhere
    • G01N33/6896—Neurological disorders, e.g. Alzheimer's disease
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; CARE OF BIRDS, FISHES, INSECTS; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00—Genetically modified animals
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; CARE OF BIRDS, FISHES, INSECTS; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00—Genetically modified animals
    • A01K2217/07—Animals genetically altered by homologous recombination
    • A01K2217/075—Animals genetically altered by homologous recombination inducing loss of function, i.e. knock out
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering N.A.
    • 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
    • C12N2320/00—Applications; Uses
    • C12N2320/10—Applications; Uses in screening processes
    • C12N2320/12—Applications; Uses in screening processes in functional genomics, i.e. for the determination of gene function
    • 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
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • 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
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/32—Special delivery means, e.g. tissue-specific
    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N2500/00—Screening for compounds of potential therapeutic value
    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N2800/00—Detection or diagnosis of diseases
    • G01N2800/16—Ophthalmology
    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N2800/00—Detection or diagnosis of diseases
    • G01N2800/28—Neurological disorders
HK05104837.4A 2002-04-18 2005-06-08 Means and methods for the specific modulation of target genes in the eye HK1072074A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
EP02008761 2002-04-18
US43117302P 2002-12-05 2002-12-05
PCT/EP2003/004003 WO2003087368A2 (fr) 2002-04-18 2003-04-16 Moyens et procedes pour la modulation specifique de genes cibles dans le snc et l'oeil, et procedes pour leur identification

Publications (1)

Publication Number Publication Date
HK1072074A1 true HK1072074A1 (en) 2005-08-12

Family

ID=47711411

Family Applications (1)

Application Number Title Priority Date Filing Date
HK05104837.4A HK1072074A1 (en) 2002-04-18 2005-06-08 Means and methods for the specific modulation of target genes in the eye

Country Status (9)

Country Link
US (5) US20050222061A1 (fr)
EP (2) EP1495121A2 (fr)
JP (2) JP2006500910A (fr)
AU (3) AU2003222820A1 (fr)
CA (2) CA2482903A1 (fr)
ES (1) ES2397060T3 (fr)
HK (1) HK1072074A1 (fr)
MX (2) MXPA04010283A (fr)
WO (2) WO2003087368A2 (fr)

Families Citing this family (49)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
MXPA04010283A (es) * 2002-04-18 2005-08-18 Acuity Pharmaceuticals Inc Medios y metodos para la modulacion especifica de genes objetivo en el cns y el ojo y metodos para su identificacion.
US7148342B2 (en) 2002-07-24 2006-12-12 The Trustees Of The University Of Pennyslvania Compositions and methods for sirna inhibition of angiogenesis
US7794704B2 (en) 2004-01-23 2010-09-14 Advanced Cell Technology, Inc. Methods for producing enriched populations of human retinal pigment epithelium cells for treatment of retinal degeneration
EP4248751A3 (fr) * 2004-01-23 2023-11-01 President and Fellows of Harvard College Modalites ameliorees du traitement des maladies degeneratives de la retine
GB0418762D0 (en) * 2004-08-23 2004-09-22 Genomica Sau Methods and compositions to treat glaucoma
CN105251024A (zh) * 2004-08-23 2016-01-20 西伦蒂斯私人股份公司 眼病的治疗
TW200639253A (en) 2005-02-01 2006-11-16 Alcon Inc RNAi-mediated inhibition of ocular targets
GB0521351D0 (en) 2005-10-20 2005-11-30 Genomica Sau Modulation of TRPV expression levels
GB0521716D0 (en) 2005-10-25 2005-11-30 Genomica Sau Modulation of 11beta-hydroxysteriod dehydrogenase 1 expression for the treatment of ocular diseases
GB0605337D0 (en) 2006-03-17 2006-04-26 Genomica Sau Treatment of CNS conditions
US20090130212A1 (en) * 2006-05-15 2009-05-21 Physical Pharmaceutica, Llc Composition and improved method for preparation of small particles
JP2009540011A (ja) * 2006-06-12 2009-11-19 エクセジェニックス、インク．ディー／ビー／エー オプコ ヘルス、インク． 血管新生のｓｉＲＮＡ阻害のための組成物及び方法
PL2056845T3 (pl) 2006-08-08 2018-01-31 Univ Bonn Rheinische Friedrich Wilhelms Struktura i zastosowanie oligonukleotydów z 5'-fosforanem
US7872118B2 (en) * 2006-09-08 2011-01-18 Opko Ophthalmics, Llc siRNA and methods of manufacture
JP2010507387A (ja) 2006-10-25 2010-03-11 クアーク・ファーマスーティカルス、インコーポレイテッド 新規のｓｉＲＮＡおよびその使用方法
CN101815521B (zh) 2007-10-03 2014-12-10 夸克制药公司 新siRNA结构
PL2209888T3 (pl) 2007-10-12 2020-05-18 Astellas Institute For Regenerative Medicine Ulepszony sposób wytwarzania komórek rpe i kompozycji komórek rpe
WO2009141146A1 (fr) 2008-05-21 2009-11-26 Gunther Hartmann Oligonucléotide à 5’-triphosphate présentant une extrémité franche et ses utilisations
US8796443B2 (en) 2008-09-22 2014-08-05 Rxi Pharmaceuticals Corporation Reduced size self-delivering RNAi compounds
NZ593743A (en) 2008-12-04 2012-07-27 Opko Ophthalmics Llc Compositions and methods for selective inhibition of pro-angiogenic vegf isoforms
WO2010080452A2 (fr) 2008-12-18 2010-07-15 Quark Pharmaceuticals, Inc. Composés d'arnsi et leurs procédés d'utilisation
WO2010090762A1 (fr) 2009-02-04 2010-08-12 Rxi Pharmaceuticals Corporation Duplexes d'arn avec régions de nucléotide phosphorothioate à brin unique pour fonctionnalité supplémentaire
KR20120102709A (ko) 2009-11-17 2012-09-18 어드밴스드 셀 테크놀로지, 인코포레이티드 인간 ｒｐｅ 세포의 생산 방법 및 인간 ｒｐｅ 세포의 제약 제제
EP2862929B1 (fr) * 2009-12-09 2017-09-06 Quark Pharmaceuticals, Inc. Compositions et procédés pour le traitement de maladies, troubles ou lésions du système nerveux central
US9080171B2 (en) 2010-03-24 2015-07-14 RXi Parmaceuticals Corporation Reduced size self-delivering RNAi compounds
WO2011119871A1 (fr) 2010-03-24 2011-09-29 Rxi Phrmaceuticals Corporation Arn interférant dans des indications oculaires
US8648053B2 (en) 2010-10-20 2014-02-11 Rosalind Franklin University Of Medicine And Science Antisense oligonucleotides that target a cryptic splice site in Ush1c as a therapeutic for Usher syndrome
WO2012085005A1 (fr) * 2010-12-23 2012-06-28 Laurent Alexandre Procédé d'obtention de moyens optiques adaptés à un individu humain souffrant ou susceptible de souffrir d'un ou plusieurs troubles ou maladies oculaires d'ordre génétique
EP2508530A1 (fr) 2011-03-28 2012-10-10 Rheinische Friedrich-Wilhelms-Universität Bonn Purification d'oligonucléotides triphosphorylés au moyen d'étiquettes de capture
US9353371B2 (en) 2011-05-02 2016-05-31 Ionis Pharmaceuticals, Inc. Antisense compounds targeting genes associated with usher syndrome
US10813630B2 (en) 2011-08-09 2020-10-27 Corquest Medical, Inc. Closure system for atrial wall
US10314594B2 (en) 2012-12-14 2019-06-11 Corquest Medical, Inc. Assembly and method for left atrial appendage occlusion
US10307167B2 (en) 2012-12-14 2019-06-04 Corquest Medical, Inc. Assembly and method for left atrial appendage occlusion
GB201215857D0 (en) 2012-09-05 2012-10-24 Sylentis Sau siRNA and their use in methods and compositions for the treatment and/or prevention of eye conditions
AU2012389270B2 (en) 2012-09-05 2018-11-08 Sylentis S.A.U. siRNA and their use in methods and compositions for the treatment and/or prevention of eye conditions
EP2712870A1 (fr) 2012-09-27 2014-04-02 Rheinische Friedrich-Wilhelms-Universität Bonn Nouveaux ligands de RIG-I et procédés pour les produire
US20140142689A1 (en) 2012-11-21 2014-05-22 Didier De Canniere Device and method of treating heart valve malfunction
US9566443B2 (en) 2013-11-26 2017-02-14 Corquest Medical, Inc. System for treating heart valve malfunction including mitral regurgitation
US10061111B2 (en) 2014-01-17 2018-08-28 The Trustees Of Columbia University In The City Of New York Systems and methods for three dimensional imaging
WO2015109323A2 (fr) * 2014-01-17 2015-07-23 The Trustees Of Columbia University In The City Of New York Systèmes et procédés d'imagerie tridimensionnelle
WO2015132303A1 (fr) 2014-03-04 2015-09-11 Sylentis Sau Arnsi et leur utilisation dans des méthodes et des compositions pour le traitement et/ou la prévention d'affections oculaires
US11279934B2 (en) 2014-04-28 2022-03-22 Phio Pharmaceuticals Corp. Methods for treating cancer using nucleic acids targeting MDM2 or MYCN
CN107073294A (zh) 2014-09-05 2017-08-18 阿克赛医药公司 使用靶向tyr或mmp1的核酸治疗老化和皮肤病症的方法
US10842626B2 (en) 2014-12-09 2020-11-24 Didier De Canniere Intracardiac device to correct mitral regurgitation
CA2978449A1 (fr) 2015-03-02 2016-09-09 180 Therapeutics Lp Methode de traitement d'un trouble fibrotique localise a l'aide d'un antagoniste de l'il-33
KR20170095417A (ko) * 2016-02-11 2017-08-23 삼성전자주식회사 발광 장치 및 전자 기기
EP3482238A1 (fr) 2016-07-10 2019-05-15 The Trustees of Columbia University in the City of New York Imagerie tridimensionnelle utilisant une excitation planaire balayée, confocale et alignée avec un relais d'images
US10712545B2 (en) * 2017-03-07 2020-07-14 The United States Of America As Represented By The Secretary, Department Of Health And Human Services Systems and methods for conducting contact-free thickness and refractive-index measurements of intraocular lenses using a self-calibrating dual confocal microscopy system
CN108739556A (zh) * 2018-08-31 2018-11-06 南京工业大学 一种光化学诱导斑马鱼缺血性脑卒中模型的应用

Family Cites Families (101)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4235871A (en) 1978-02-24 1980-11-25 Papahadjopoulos Demetrios P Method of encapsulating biologically active materials in lipid vesicles
US4501728A (en) * 1983-01-06 1985-02-26 Technology Unlimited, Inc. Masking of liposomes from RES recognition
US5019369A (en) * 1984-10-22 1991-05-28 Vestar, Inc. Method of targeting tumors in humans
US5545412A (en) * 1985-01-07 1996-08-13 Syntex (U.S.A.) Inc. N-[1, (1-1)-dialkyloxy]-and N-[1, (1-1)-dialkenyloxy]-alk-1-yl-n,n,n-tetrasubstituted ammonium lipids and uses therefor
US5139941A (en) * 1985-10-31 1992-08-18 University Of Florida Research Foundation, Inc. AAV transduction vectors
US4920016A (en) * 1986-12-24 1990-04-24 Linear Technology, Inc. Liposomes with enhanced circulation time
US4837028A (en) * 1986-12-24 1989-06-06 Liposome Technology, Inc. Liposomes with enhanced circulation time
IE72103B1 (en) * 1987-08-12 1997-03-12 Hem Res Inc Promotion of host defense by systemic dsRNA treatment
US5683986A (en) * 1987-08-12 1997-11-04 Hemispherx Biopharma Inc. Elaboration of host defense mediators into biological fluids by systemic dsRNA treatment
US5712257A (en) * 1987-08-12 1998-01-27 Hem Research, Inc. Topically active compositions of mismatched dsRNAs
US5498521A (en) * 1990-01-24 1996-03-12 President And Fellows Of Harvard College Diagnosis of hereditary retinal degenerative diseases
US5843738A (en) * 1990-08-14 1998-12-01 Isis Pharmaceuticals, Inc. Oligonucleotide modulation of cell adhesion
US5252479A (en) * 1991-11-08 1993-10-12 Research Corporation Technologies, Inc. Safe vector for gene therapy
US5587308A (en) 1992-06-02 1996-12-24 The United States Of America As Represented By The Department Of Health & Human Services Modified adeno-associated virus vector capable of expression from a novel promoter
ATE404683T1 (de) 1992-09-25 2008-08-15 Aventis Pharma Sa Adenovirus vektoren für die übertragung fremder gene in zellen des zentralen nervensystems, insbesondere im gehirn
US6177401B1 (en) * 1992-11-13 2001-01-23 Max-Planck-Gesellschaft Zur Forderung Der Wissenschaften Use of organic compounds for the inhibition of Flk-1 mediated vasculogenesis and angiogenesis
US5478745A (en) 1992-12-04 1995-12-26 University Of Pittsburgh Recombinant viral vector system
GB9308271D0 (en) 1993-04-21 1993-06-02 Univ Edinburgh Method of isolating and/or enriching and/or selectively propagating pluripotential animal cells and animals for use in said method
US5731294A (en) * 1993-07-27 1998-03-24 Hybridon, Inc. Inhibition of neovasularization using VEGF-specific oligonucleotides
US5639872A (en) * 1993-07-27 1997-06-17 Hybridon, Inc. Human VEGF-specific oligonucleotides
US6410322B1 (en) 1993-07-27 2002-06-25 Hybridon Inc Antisense oligonucleotide inhibition of vascular endothelial growth factor expression
US5624803A (en) * 1993-10-14 1997-04-29 The Regents Of The University Of California In vivo oligonucleotide generator, and methods of testing the binding affinity of triplex forming oligonucleotides derived therefrom
US6037329A (en) * 1994-03-15 2000-03-14 Selective Genetics, Inc. Compositions containing nucleic acids and ligands for therapeutic treatment
US6150092A (en) * 1994-06-27 2000-11-21 Taogosei Company, Ltd. Antisense nucleic acid compound targeted to VEGF
US5882914A (en) * 1995-06-06 1999-03-16 The Johns Hopkins University School Of Medicine Nucleic acids encoding the hypoxia inducible factor-1
US20030216335A1 (en) * 2001-11-30 2003-11-20 Jennifer Lockridge Method and reagent for the modulation of female reproductive diseases and conditions
WO1997018855A1 (fr) 1995-11-21 1997-05-29 Eduard Naumovich Lerner Dispositif pour ameliorer l'administration a l'organisme de substances et de composes biologiquement actifs
GB9524807D0 (en) 1995-12-05 1996-02-07 Smithkline Beecham Plc Novel compounds
US5843016A (en) * 1996-03-18 1998-12-01 Physion S.R.L. Electromotive drug administration for treatment of acute urinary outflow obstruction
US7033598B2 (en) * 1996-11-19 2006-04-25 Intrabrain International N.V. Methods and apparatus for enhanced and controlled delivery of a biologically active agent into the central nervous system of a mammal
WO1998022132A1 (fr) 1996-11-20 1998-05-28 Yissum Res Dev Co Methode et composition permettant le passage a travers la barriere hemato-encephalique
US6165709A (en) * 1997-02-28 2000-12-26 Fred Hutchinson Cancer Research Center Methods for drug target screening
US20050096282A1 (en) * 1997-04-21 2005-05-05 Lewin Alfred S. Adeno-associated virus-delivered ribozyme compositions and methods for the treatment of retinal diseases
NZ500427A (en) 1997-04-21 2002-12-20 Univ Florida The use of ribozyme treatment for retinal diseases
US7025991B2 (en) 1997-06-13 2006-04-11 Nanodel Technologies Gmbh Drug targeting system, method of its preparation and its use
US5902598A (en) * 1997-08-28 1999-05-11 Control Delivery Systems, Inc. Sustained release drug delivery devices
AU9040098A (en) 1997-09-10 1999-03-29 University Of Florida Compounds and method for the prevention and treatment of diabetic retinopathy
US6506559B1 (en) * 1997-12-23 2003-01-14 Carnegie Institute Of Washington Genetic inhibition by double-stranded RNA
US20030096775A1 (en) 2001-10-23 2003-05-22 Isis Pharmaceuticals Inc. Antisense modulation of complement component C3 expression
US6433145B1 (en) * 1998-07-21 2002-08-13 Human Genome Sciences, Inc. Keratinocyte derived interferon
EP0979869A1 (fr) * 1998-08-07 2000-02-16 Hoechst Marion Roussel Deutschland GmbH Oligonucléotides courts pour inhiber l'expression de VEGF
US6219557B1 (en) * 1998-12-11 2001-04-17 Ericsson Inc. System and method for providing location services in parallel to existing services in general packet radio services architecture
AU776150B2 (en) * 1999-01-28 2004-08-26 Medical College Of Georgia Research Institute, Inc. Composition and method for (in vivo) and (in vitro) attenuation of gene expression using double stranded RNA
DE19956568A1 (de) 1999-01-30 2000-08-17 Roland Kreutzer Verfahren und Medikament zur Hemmung der Expression eines vorgegebenen Gens
DK1150918T3 (da) * 1999-02-03 2004-12-20 Biosante Pharmaceuticals Inc Fremgangsmåde til fremstilling af terapeutiske calciumphosphatpartikler
US6121000A (en) * 1999-02-11 2000-09-19 Genesense Technologies, Inc. Antitumor antisense sequences directed against R1 and R2 components of ribonucleotide reductase
WO2000063364A2 (fr) * 1999-04-21 2000-10-26 American Home Products Corporation Procedes et compositions pour l'inhibition de la fonction de sequences polynucleotidiques
US6331313B1 (en) * 1999-10-22 2001-12-18 Oculex Pharmaceticals, Inc. Controlled-release biocompatible ocular drug delivery implant devices and methods
GB9927444D0 (en) 1999-11-19 2000-01-19 Cancer Res Campaign Tech Inhibiting gene expression
EP2944695B1 (fr) * 1999-12-16 2017-02-15 Monsanto Technology LLC Nouvelles constructions d'expréssion végétales
CA2397919A1 (fr) 2000-01-19 2001-07-26 Parkash S. Gill Compositions renfermant des oligonucleotides antisens diriges contre le vegf et methodes associees
AU3974001A (en) 2000-02-03 2001-08-14 Ali R. Fattaey Method and reagent for the inhibition of checkpoint kinase-1 (chk 1) enzyme
JP2003526367A (ja) 2000-03-16 2003-09-09 ジェネティカ インコーポレイテッド Ｒｎａ干渉の方法とｒｎａ干渉組成物
CA2404890C (fr) 2000-03-30 2013-11-19 Whitehead Institute For Biomedical Research Mediateurs d'interference arn specifiques de sequences arn
US6372250B1 (en) * 2000-04-25 2002-04-16 The Regents Of The University Of California Non-invasive gene targeting to the brain
US6375972B1 (en) * 2000-04-26 2002-04-23 Control Delivery Systems, Inc. Sustained release drug delivery devices, methods of use, and methods of manufacturing thereof
WO2001083729A2 (fr) * 2000-05-01 2001-11-08 Novartis Ag Vecteurs pour transduction oculaire et utilisation en therapie genique
JP2004520261A (ja) 2000-06-23 2004-07-08 イシウム リサーチ デベロップメント カンパニー オブ ザ ヘブリュー ユニバーシティー オブ イエルサレム 改善された性質を有する正電荷を有するペプチド核酸類縁体
AU2001271614B2 (en) * 2000-07-03 2007-05-31 Catalent Pharma Solutions, Llc Host cells containing multiple integrating vectors
JP2004520006A (ja) * 2000-07-21 2004-07-08 ザ トラスティース オブ コロンビア ユニバーシティ インザ シティ オブ ニューヨーク ラットｐｅｇ−３プロモーターの領域を含んでなる核酸とその利用
IL137672A0 (en) 2000-08-03 2001-10-31 Dpharm Ltd Derivatives of branched-chain lipophilic molecules and uses thereof
US6443145B1 (en) * 2000-08-25 2002-09-03 Learning Legacy Solar seeker
US20020132788A1 (en) * 2000-11-06 2002-09-19 David Lewis Inhibition of gene expression by delivery of small interfering RNA to post-embryonic animal cells in vivo
US20020173478A1 (en) * 2000-11-14 2002-11-21 The Trustees Of The University Of Pennsylvania Post-transcriptional gene silencing by RNAi in mammalian cells
BRPI0115814B8 (pt) * 2000-12-01 2021-05-25 Europaeisches Laboratorium Fuer Molekularbiologie Embl moléculas de rna de filamento duplo, seu método de preparação e composição farmacêutica compreendendo as mesmas
EP1229134A3 (fr) 2001-01-31 2004-01-28 Nucleonics, Inc Utilisation de l'inhibition post-transcriptionnelle pour l'identification des séquences d'acides nucléiques qui modifient la fonction d'une cellule
US20020165158A1 (en) * 2001-03-27 2002-11-07 King George L. Methods of modulating angiogenesis
JP2002305193A (ja) * 2001-04-05 2002-10-18 Sony Corp 半導体装置とその製造方法
US20030045830A1 (en) 2001-04-17 2003-03-06 De Bizemont Therese Gene therapy with chimeric oligonucleotides delivered by a method comprising a step of iontophoresis
WO2003070910A2 (fr) 2002-02-20 2003-08-28 Ribozyme Pharmaceuticals, Incorporated Inhibition induite par interference d'arn du facteur de croissance endothelial vasculaire et expression genetique du recepteur de facteur de croissance endothelial vasculaire au moyen d'acides nucleiques interferents courts (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)
US20050159380A1 (en) * 2001-05-18 2005-07-21 Sirna Therapeutics, Inc. RNA interference mediated inhibition of angiopoietin gene expression using short interfering nucleic acid (siNA)
US7517864B2 (en) * 2001-05-18 2009-04-14 Sirna Therapeutics, Inc. RNA interference mediated inhibition of vascular endothelial growth factor and vascular endothelial growth factor receptor gene expression using short interfering nucleic acid (siNA)
US20050187174A1 (en) * 2001-05-18 2005-08-25 Sirna Therapeutics, Inc. RNA interference mediated inhibition of intercellular adhesion molecule (ICAM) gene expression using short interfering nucleic acid (siNA)
US20050148530A1 (en) 2002-02-20 2005-07-07 Sirna Therapeutics, Inc. RNA interference mediated inhibition of vascular endothelial growth factor and vascular endothelial growth factor receptor gene expression using short interfering nucleic acid (siNA)
CA2448320A1 (fr) 2001-05-29 2002-12-05 Sirna Therapeutics, Inc. Modulation a base de l'acide nucleique des maladies et troubles de l'appareil reproducteur chez la femme
US20030087755A1 (en) 2001-05-29 2003-05-08 Linfeng Chen Olefin polymerization catalyst compositions and method of preparation
EP1409506B1 (fr) * 2001-07-23 2012-05-09 The Board Of Trustees Of The Leland Stanford Junior University Methodes et compositions pour l'inhibition induite par l'arni de l'expression genetique chez des mammiferes
US7820178B2 (en) 2001-08-01 2010-10-26 University of Brisol VEGF isoforms and their use as anti-angiogenic, anti-vasodilatory, anti-permeability and anti-proliferative agents
AU2002348163A1 (en) * 2001-11-02 2003-05-19 Intradigm Corporation Therapeutic methods for nucleic acid delivery vehicles
WO2003046173A1 (fr) * 2001-11-28 2003-06-05 Center For Advanced Science And Technology Incubation, Ltd. Systeme d'expression d'arn si et procede de production de cellules d'inactivation de genes fonctionnelles et analogues au moyen de ce systeme
HUE037352T2 (hu) * 2002-04-05 2018-08-28 Roche Innovation Ct Copenhagen As A HIF-1alfa expresszálódását módosító oligomer vegyületek
MXPA04010283A (es) 2002-04-18 2005-08-18 Acuity Pharmaceuticals Inc Medios y metodos para la modulacion especifica de genes objetivo en el cns y el ojo y metodos para su identificacion.
AU2003237686A1 (en) 2002-05-24 2003-12-12 Max-Planck Gesellschaft Zur Forderung Der Wissenschaften E.V. Rna interference mediating small rna molecules
US20040115640A1 (en) * 2002-12-11 2004-06-17 Isis Pharmaceuticals Inc. Modulation of angiopoietin-2 expression
US7148342B2 (en) * 2002-07-24 2006-12-12 The Trustees Of The University Of Pennyslvania Compositions and methods for sirna inhibition of angiogenesis
US20060211637A1 (en) 2002-08-06 2006-09-21 Intradigm Corporation Methods of down regulating target gene expression in vivo by introduction of interfering rna
JP5449639B2 (ja) * 2002-11-01 2014-03-19 ザ トラスティーズ オブ ザ ユニバーシティ オブ ペンシルバニア ＨＩＦ−１アルファのｓｉＲＮＡ阻害に関する組成物及び方法
JP2006507841A (ja) * 2002-11-14 2006-03-09 ダーマコン， インコーポレイテッド 機能的ｓｉＲＮＡおよび超機能的ｓｉＲＮＡ
EP1604010B1 (fr) * 2003-01-16 2010-08-11 The Trustees of The University of Pennsylvania Compositions et procedes destines a l'inhibition du gene icam-1 par arn a faible inhibition
AU2004233043A1 (en) * 2003-04-18 2004-11-04 The Trustees Of The University Of Pennsylvania Compositions and methods for siRNA inhibition of angiopoietin 1 and 2 and their receptor Tie2
US20050019927A1 (en) * 2003-07-13 2005-01-27 Markus Hildinger DECREASING GENE EXPRESSION IN A MAMMALIAN SUBJECT IN VIVO VIA AAV-MEDIATED RNAi EXPRESSION CASSETTE TRANSFER
EP1735009A4 (fr) * 2004-03-12 2011-03-30 Alnylam Pharmaceuticals Inc Agents arni ciblant le facteur de croissance de l'endothelium vasculaire (vegf)
US20060182783A1 (en) * 2004-04-30 2006-08-17 Allergan, Inc. Sustained release intraocular drug delivery systems
TWI243957B (en) * 2004-08-16 2005-11-21 Asia Optical Co Inc Assembling method of reticle module
BRPI0610499A2 (pt) 2005-04-12 2010-06-22 Intradigm Corp moléculas de ácido nucléico , composições e usos das referidas moléculas
DE602006014026D1 (de) * 2005-12-22 2010-06-10 Opko Ophthalmics Llc Zusammensetzungen und verfahren zur regulierung eines komplementsystems
EP1982176A1 (fr) * 2006-01-30 2008-10-22 Dako Denmark A/S Quantification ultra-rapide de lymphocytes-t specifiques d'antigenes dans du sang entier par cytometrie de flux
JP2009540011A (ja) 2006-06-12 2009-11-19 エクセジェニックス、インク．ディー／ビー／エー オプコ ヘルス、インク． 血管新生のｓｉＲＮＡ阻害のための組成物及び方法
US7872118B2 (en) 2006-09-08 2011-01-18 Opko Ophthalmics, Llc siRNA and methods of manufacture
GB0704678D0 (en) 2007-03-09 2007-04-18 Univ Bristol Pro- and anti-angiogenic treatments

Also Published As

Publication number Publication date
US20150105448A1 (en) 2015-04-16
EP1495121A2 (fr) 2005-01-12
MXPA04010283A (es) 2005-08-18
JP2005538940A (ja) 2005-12-22
MXPA04010282A (es) 2005-08-18
WO2003087367A3 (fr) 2004-04-29
US8202845B2 (en) 2012-06-19
JP5578388B2 (ja) 2014-08-27
US8946180B2 (en) 2015-02-03
JP2006500910A (ja) 2006-01-12
WO2003087367A2 (fr) 2003-10-23
US20050222061A1 (en) 2005-10-06
AU2003224087B2 (en) 2009-03-05
US20060003915A1 (en) 2006-01-05
EP1495120B1 (fr) 2012-10-10
EP1495120A2 (fr) 2005-01-12
AU2003222820A1 (en) 2003-10-27
AU2009200699A1 (en) 2009-03-12
CA2482903A1 (fr) 2003-10-23
CA2482904A1 (fr) 2003-10-23
WO2003087368A2 (fr) 2003-10-23
WO2003087368A3 (fr) 2004-04-29
AU2003224087A1 (en) 2003-10-27
ES2397060T3 (es) 2013-03-04
US20120277288A1 (en) 2012-11-01
US20110021605A1 (en) 2011-01-27

Similar Documents

Publication Publication Date Title
HK1072074A1 (en) Means and methods for the specific modulation of target genes in the eye
AU2003286854A1 (en) Methods and apparatuses for performing procedures in the subarachnoid space
AU2003210890A8 (en) Controlled lighting methods and apparatus
EP1501544A4 (fr) Alcam et modulateurs d'alcam
EP1539931A4 (fr) Agents de modulation de la differenciation et utilisations associees
EP1467916A4 (fr) Conteneur et procedes associes
IL175509A0 (en) Methods and agents for immune modulation
AU2003300420A1 (en) Material heat treatment system and method
AU2003259285A8 (en) Methods of identifying adipocyte specific genes, the genes identified, and their uses
EP1490074A4 (fr) Therapie genique cellulaire mixte
AUPS051002A0 (en) Low level laser therapy method and means
GB0308382D0 (en) Therapeutic methods and means
EP1497428A4 (fr) Technique de transfection et d'expression de surface
AU2003269914A8 (en) Kit and method in eye surgery
EP1731610A4 (fr) Proteines conferant une tolerance vis-a-vis du bore et genes de celles-ci
AU2003216354A8 (en) Modulators of paraptosis and related methods
AU2003239589A8 (en) Eta-1 gene and methods for use
AU2003285490A8 (en) Astrocyte modulated genes and uses thereof
EP1702072A4 (fr) Therapie genique et modification genetique mettant en oeuvre des minichromosomes neocentromeriques
AU2003287943A8 (en) Biogeometry shapes and methodology for biological energy balancing
GB0223860D0 (en) Polypeptide methods and means
GB0225663D0 (en) Methods and means for modulating the immune response
GB0327844D0 (en) Therapeutic methods and means
GB0209039D0 (en) Therapeutic methods and means
GB0216754D0 (en) Therapeutic methods and means