AU2003222820A1 - Means and methods for the specific modulation of target genes in the cns and the eye and methods for their identification - Google Patents
Means and methods for the specific modulation of target genes in the cns and the eye and methods for their identification Download PDFInfo
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- AU2003222820A1 AU2003222820A1 AU2003222820A AU2003222820A AU2003222820A1 AU 2003222820 A1 AU2003222820 A1 AU 2003222820A1 AU 2003222820 A AU2003222820 A AU 2003222820A AU 2003222820 A AU2003222820 A AU 2003222820A AU 2003222820 A1 AU2003222820 A1 AU 2003222820A1
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- 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
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- A61K31/7088—Compounds having three or more nucleosides or nucleotides
- A61K31/713—Double-stranded nucleic acids or oligonucleotides
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- 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
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- 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
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- 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
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- G—PHYSICS
- G01—MEASURING; TESTING
- G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
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- 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
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- 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
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- 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
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- A—HUMAN NECESSITIES
- A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
- A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
- A01K2217/00—Genetically modified animals
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- A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; 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
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- C12N2310/10—Type of nucleic acid
- C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
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- C12N2320/12—Applications; Uses in screening processes in functional genomics, i.e. for the determination of gene function
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- Wood Science & Technology (AREA)
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- Microbiology (AREA)
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- Hematology (AREA)
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Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| AU2009200699A AU2009200699A1 (en) | 2002-04-18 | 2009-02-23 | Means and methods for the specific modulation of target genes in the CNS and the eye and methods for their identification |
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| EP02008761 | 2002-04-18 | ||
| EP02008761.5 | 2002-04-18 | ||
| US43117302P | 2002-12-05 | 2002-12-05 | |
| US60/431,173 | 2002-12-05 | ||
| PCT/EP2003/004003 WO2003087368A2 (en) | 2002-04-18 | 2003-04-16 | Means and methods for the specific modulation of target genes in the cns and the eye and methods for their identification |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| AU2009200699A Division AU2009200699A1 (en) | 2002-04-18 | 2009-02-23 | Means and methods for the specific modulation of target genes in the CNS and the eye and methods for their identification |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| AU2003222820A1 true AU2003222820A1 (en) | 2003-10-27 |
Family
ID=47711411
Family Applications (3)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| AU2003222820A Abandoned AU2003222820A1 (en) | 2002-04-18 | 2003-04-16 | Means and methods for the specific modulation of target genes in the cns and the eye and methods for their identification |
| AU2003224087A Ceased AU2003224087B2 (en) | 2002-04-18 | 2003-04-16 | Means and methods for the specific inhibition of genes in cells and tissue of the CNS and/or eye |
| AU2009200699A Abandoned AU2009200699A1 (en) | 2002-04-18 | 2009-02-23 | Means and methods for the specific modulation of target genes in the CNS and the eye and methods for their identification |
Family Applications After (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| AU2003224087A Ceased AU2003224087B2 (en) | 2002-04-18 | 2003-04-16 | Means and methods for the specific inhibition of genes in cells and tissue of the CNS and/or eye |
| AU2009200699A Abandoned AU2009200699A1 (en) | 2002-04-18 | 2009-02-23 | Means and methods for the specific modulation of target genes in the CNS and the eye and methods for their identification |
Country Status (8)
| Country | Link |
|---|---|
| US (5) | US20050222061A1 (cg-RX-API-DMAC10.html) |
| EP (2) | EP1495120B1 (cg-RX-API-DMAC10.html) |
| JP (2) | JP2006500910A (cg-RX-API-DMAC10.html) |
| AU (3) | AU2003222820A1 (cg-RX-API-DMAC10.html) |
| CA (2) | CA2482903A1 (cg-RX-API-DMAC10.html) |
| ES (1) | ES2397060T3 (cg-RX-API-DMAC10.html) |
| MX (2) | MXPA04010282A (cg-RX-API-DMAC10.html) |
| WO (2) | WO2003087367A2 (cg-RX-API-DMAC10.html) |
Families Citing this family (51)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US20050222061A1 (en) | 2002-04-18 | 2005-10-06 | Schulte Ralf W | Means and methods for the specific inhibition of genes in cells and tissue of the cns and/or eye |
| 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 |
| CA2937099A1 (en) * | 2004-01-23 | 2005-08-04 | Astellas Institute For Regenerative Medicine | Improved modalities for the treatment of degenerative diseases of the retina |
| GB0418762D0 (en) * | 2004-08-23 | 2004-09-22 | Genomica Sau | Methods and compositions to treat glaucoma |
| DK1781787T3 (en) | 2004-08-23 | 2017-07-31 | Sylentis Sau | TREATMENT OF EYE DISORDERS FEATURED BY AN INCREASED INTRAOCULAR PRESSURE WITH SIRNAS |
| 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 |
| JP2010500011A (ja) | 2006-08-08 | 2010-01-07 | ガンサー ハートマン | 5’リン酸オリゴヌクレオチドの構造および使用 |
| 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 |
| CA2701845A1 (en) | 2007-10-03 | 2009-04-09 | Quark Pharmaceuticals, Inc. | Novel sirna structures |
| EP2209888B1 (en) | 2007-10-12 | 2019-12-04 | Astellas Institute for Regenerative Medicine | Improved methods of producing rpe cells and compositions of rpe cells |
| US9738680B2 (en) | 2008-05-21 | 2017-08-22 | Rheinische Friedrich-Wilhelms-Universität Bonn | 5′ triphosphate oligonucleotide with blunt end and uses thereof |
| EP2342340A1 (en) | 2008-09-22 | 2011-07-13 | Rxi Pharmaceuticals Corporation | Rna interference in skin indications |
| JP5832293B2 (ja) | 2008-12-04 | 2015-12-16 | オプコ ファーマシューティカルズ、エルエルシー | 血管新生促進vegfイソ型を選択的に抑制する組成物および方法 |
| US20110288155A1 (en) | 2008-12-18 | 2011-11-24 | Elena Feinstein | Sirna compounds and methods of use thereof |
| WO2010090762A1 (en) | 2009-02-04 | 2010-08-12 | Rxi Pharmaceuticals Corporation | Rna duplexes with single stranded phosphorothioate nucleotide regions for additional functionality |
| IL281453B (en) | 2009-11-17 | 2022-07-01 | Astellas Inst For Regenerative Medicine | Methods for preparing human rpe cells and pharmaceutical preparations of human rpe cells |
| WO2011072091A1 (en) * | 2009-12-09 | 2011-06-16 | Quark Pharmaceuticals, Inc. | Methods and compositions for treating diseases, disorders or injury of the cns |
| WO2011119852A1 (en) | 2010-03-24 | 2011-09-29 | Rxi Pharmaceuticals Corporation | Reduced size self-delivering rnai compounds |
| US9095504B2 (en) | 2010-03-24 | 2015-08-04 | Rxi Pharmaceuticals Corporation | RNA interference in ocular indications |
| 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 |
| CA2822261A1 (en) * | 2010-12-23 | 2012-06-28 | Laurent Alexandre | Method to obtain optical means adapted to a human individual suffering or susceptible to suffer from one or more genetic related eye disorder(s) or disease(s) |
| EP2508530A1 (en) | 2011-03-28 | 2012-10-10 | Rheinische Friedrich-Wilhelms-Universität Bonn | Purification of triphosphorylated oligonucleotides using capture tags |
| WO2012151324A1 (en) * | 2011-05-02 | 2012-11-08 | Isis Pharmaceuticals, Inc. | Antisense compounds targeting genes associated with usher syndrome |
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- 2003-04-16 US US10/511,656 patent/US20050222061A1/en not_active Abandoned
- 2003-04-16 JP JP2003584309A patent/JP2006500910A/ja active Pending
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- 2003-04-16 EP EP03718764A patent/EP1495120B1/en not_active Expired - Lifetime
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| US8202845B2 (en) | 2012-06-19 |
| JP5578388B2 (ja) | 2014-08-27 |
| US8946180B2 (en) | 2015-02-03 |
| CA2482904A1 (en) | 2003-10-23 |
| US20050222061A1 (en) | 2005-10-06 |
| AU2003224087A1 (en) | 2003-10-27 |
| JP2005538940A (ja) | 2005-12-22 |
| AU2003224087B2 (en) | 2009-03-05 |
| WO2003087368A3 (en) | 2004-04-29 |
| CA2482903A1 (en) | 2003-10-23 |
| EP1495120B1 (en) | 2012-10-10 |
| ES2397060T3 (es) | 2013-03-04 |
| JP2006500910A (ja) | 2006-01-12 |
| EP1495120A2 (en) | 2005-01-12 |
| US20120277288A1 (en) | 2012-11-01 |
| HK1072074A1 (en) | 2005-08-12 |
| AU2009200699A1 (en) | 2009-03-12 |
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