ATE439440T1 - Sirna-vermitteltes gen-silencing von alpha- synuklein - Google Patents
Sirna-vermitteltes gen-silencing von alpha- synukleinInfo
- Publication number
- ATE439440T1 ATE439440T1 AT05816006T AT05816006T ATE439440T1 AT E439440 T1 ATE439440 T1 AT E439440T1 AT 05816006 T AT05816006 T AT 05816006T AT 05816006 T AT05816006 T AT 05816006T AT E439440 T1 ATE439440 T1 AT E439440T1
- Authority
- AT
- Austria
- Prior art keywords
- synuklein
- sirna
- alpha
- gene silencing
- mediated gene
- Prior art date
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
-
- 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
- A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
- A61P25/16—Anti-Parkinson drugs
-
- 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
- C12N2310/11—Antisense
- C12N2310/111—Antisense spanning the whole gene, or a large part of it
-
- 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 nucleic acids [NA]
-
- 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
- C12N2310/50—Physical structure
- C12N2310/53—Physical structure partially self-complementary or closed
Landscapes
- Health & Medical Sciences (AREA)
- Engineering & Computer Science (AREA)
- Life Sciences & Earth Sciences (AREA)
- Biomedical Technology (AREA)
- Genetics & Genomics (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Chemical & Material Sciences (AREA)
- Organic Chemistry (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- General Health & Medical Sciences (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Molecular Biology (AREA)
- Neurology (AREA)
- Neurosurgery (AREA)
- Biochemistry (AREA)
- Veterinary Medicine (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Microbiology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Medicinal Chemistry (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Psychology (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Saccharide Compounds (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US61411204P | 2004-09-29 | 2004-09-29 | |
| PCT/US2005/034516 WO2006039253A2 (en) | 2004-09-29 | 2005-09-27 | Sirna-mediated gene silencing of alpha synuclein |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| ATE439440T1 true ATE439440T1 (de) | 2009-08-15 |
Family
ID=36142994
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| AT05816006T ATE439440T1 (de) | 2004-09-29 | 2005-09-27 | Sirna-vermitteltes gen-silencing von alpha- synuklein |
Country Status (6)
| Country | Link |
|---|---|
| US (2) | US20070172462A1 (de) |
| EP (1) | EP1799826B1 (de) |
| AT (1) | ATE439440T1 (de) |
| CA (1) | CA2580189C (de) |
| DE (1) | DE602005015994D1 (de) |
| WO (1) | WO2006039253A2 (de) |
Families Citing this family (42)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2004060403A2 (en) * | 2003-01-06 | 2004-07-22 | Angiochem Inc. | Aprotinin and anglos as carriers across the blood-brain barrier |
| US20090016959A1 (en) * | 2005-02-18 | 2009-01-15 | Richard Beliveau | Delivery of antibodies to the central nervous system |
| RU2408605C2 (ru) * | 2005-02-18 | 2011-01-10 | Анджиокем Инк. | Полипептид, способный преодолевать гематоэнцефалический барьер, и его конъюгат |
| ES2744225T3 (es) | 2005-07-15 | 2020-02-24 | Angiochem Inc | Uso de polipéptidos de aprotinina como portadores en conjugados farmacéuticos |
| US20090010894A1 (en) * | 2005-12-23 | 2009-01-08 | The Parkinson's Institute | Methods and systems for identifying compounds that modulate alpha-synuclein aggregation |
| US7897568B2 (en) * | 2006-03-03 | 2011-03-01 | Vinay K. Singh | Compositions for treatment of cancer |
| GB0610183D0 (en) * | 2006-05-23 | 2006-06-28 | Isis Innovation | Treatment of neurodegenerative diseases |
| WO2008033285A2 (en) * | 2006-09-15 | 2008-03-20 | The Trustees Of Culumbia University In The City Of New York | Delivery of double-stranded rna into the central nervous system |
| WO2008109509A1 (en) * | 2007-03-02 | 2008-09-12 | Mdrna, Inc. | Nucleic acid compounds for inhibiting snca gene expression and uses thereof |
| US9365634B2 (en) * | 2007-05-29 | 2016-06-14 | Angiochem Inc. | Aprotinin-like polypeptides for delivering agents conjugated thereto to tissues |
| GB0715809D0 (en) * | 2007-08-14 | 2007-09-26 | Univ Leuven Kath | Alpha synuclein toxicity |
| MX355683B (es) * | 2008-04-18 | 2018-04-26 | Angiochem Inc | Composiciones farmacéuticas de paclitaxel, análogos de paclitaxel o conjugados de paclitaxel, y métodos relacionados de preparación y uso. |
| CA2740316A1 (en) | 2008-10-15 | 2010-04-22 | Angiochem Inc. | Conjugates of glp-1 agonists and uses thereof |
| WO2010043049A1 (en) | 2008-10-15 | 2010-04-22 | Angiochem Inc. | Etoposide and doxorubicin conjugates for drug delivery |
| CA2745524C (en) * | 2008-12-05 | 2020-06-09 | Angiochem Inc. | Conjugates of neurotensin or neurotensin analogs and uses thereof |
| US8853353B2 (en) | 2008-12-17 | 2014-10-07 | Angiochem, Inc. | Membrane type-1 matrix metalloprotein inhibitors and uses thereof |
| MX2011011023A (es) | 2009-04-20 | 2012-01-20 | Angiochem Inc | Tratamiento de cancer de ovarios usando un agente anti-cancer conjugado con un analogo de angiopep-2. |
| US9255266B2 (en) | 2009-05-06 | 2016-02-09 | Rutgers, The State University Of New Jersey | RNA targeting in alpha-synucleinopathies |
| IT1398977B1 (it) | 2009-06-25 | 2013-03-28 | Icgeb | Agente anticancro specifico per i tumori cerebrali con meccanismo di soppressione di recq1 |
| RU2012103240A (ru) | 2009-07-02 | 2013-08-10 | Ангиокем Инк. | Мультимерные пептидные конъюгаты и их применение |
| AU2010305284A1 (en) * | 2009-10-06 | 2012-05-03 | Angiochem Inc. | Compositions and methods for the transport of therapeutic agents |
| EP2380595A1 (de) | 2010-04-19 | 2011-10-26 | Nlife Therapeutics S.L. | Zusammensetzungen und Verfahren zur selektiven Abgabe von Oligonukleotidmolekülen an spezifische Neuronentypen |
| CA2817960C (en) | 2010-11-17 | 2020-06-09 | Ionis Pharmaceuticals, Inc. | Modulation of alpha synuclein expression |
| EP2721145A4 (de) * | 2011-06-16 | 2014-12-10 | Parkinson S Inst | Screening mit hohem durchsatz in hautfibroblasten mit einer alpha-synuklein-triplikation |
| HK1202434A1 (en) * | 2011-11-22 | 2015-10-02 | The Children's Hospital Of Philadelphia | Virus vectors for highly efficient transgene delivery |
| IL297919A (en) | 2013-07-22 | 2023-01-01 | Childrens Hospital Philadelphia | Variant aav and compositions, methods and uses for gene transfer to cells, organs and tissues |
| EP3091087A1 (de) * | 2015-05-08 | 2016-11-09 | Fundació Institut d'Investigació en Ciències de la Salut Germans Trias i Pujol | Verfahren zur in-vitro-diagnose von synukleinopathien mit alpha-synuklein-gentranskripten |
| EP3307326B9 (de) | 2015-06-15 | 2021-02-24 | Angiochem Inc. | Verfahren zur behandlung von leptomeningealer karzinomatose |
| MX390848B (es) | 2015-06-23 | 2025-03-21 | Childrens Hospital Philadelphia | Factor ix modificado, y composiciones, metodos y usos para la transferencia de genes a celulas, organos y tejidos. |
| WO2018090054A1 (en) | 2016-11-14 | 2018-05-17 | Berg Llc | Methods for treating parkinson's disease |
| CN112501208A (zh) | 2017-10-03 | 2021-03-16 | 普利维尔治疗公司 | 用于溶酶体障碍的基因疗法 |
| BR112020006661A2 (pt) | 2017-10-03 | 2020-10-13 | Prevail Therapeutics, Inc. | terapias de genes para distúrbios lipossomais |
| AU2018346104B2 (en) | 2017-10-03 | 2023-07-27 | Prevail Therapeutics, Inc. | Gene therapies for lysosomal disorders |
| TWI809004B (zh) | 2017-11-09 | 2023-07-21 | 美商Ionis製藥公司 | 用於降低snca表現之化合物及方法 |
| SG11202006142PA (en) | 2018-01-12 | 2020-07-29 | Roche Innovation Ct Copenhagen As | Alpha-synuclein antisense oligonucleotides and uses thereof |
| SG11202006528XA (en) | 2018-01-12 | 2020-08-28 | Bristol Myers Squibb Co | Antisense oligonucleotides targeting alpha-synuclein and uses thereof |
| CN120310791A (zh) | 2018-01-12 | 2025-07-15 | 百时美施贵宝公司 | 靶向α-突触核蛋白的反义寡核苷酸及其用途 |
| NL2022538B1 (en) * | 2019-02-08 | 2020-08-19 | Academisch Ziekenhuis Groningen | Methods for providing purified viral particles of Semliki Forest Virus (SFV), preparations obtainable thereby, and uses thereof. |
| JP7664848B2 (ja) | 2019-04-10 | 2025-04-18 | プリベイル セラピューティクス，インコーポレーテッド | リソソーム障害のための遺伝子療法 |
| WO2022268835A1 (en) * | 2021-06-21 | 2022-12-29 | Uniqure Biopharma B.V. | Gene constructs for silencing alpha-synuclein and uses thereof |
| EP4342988A1 (de) | 2022-09-20 | 2024-03-27 | Eberhard Karls Universität Tübingen, Medizinische Fakultät | Zusammensetzungen und verfahren zur modifikation der expression eines snca-gens |
| WO2025085713A1 (en) * | 2023-10-20 | 2025-04-24 | Spark Therapeutics, Inc. | Inhibitory nucleic acid targeting alpha synuclein expression |
Family Cites Families (9)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| AUPP249298A0 (en) * | 1998-03-20 | 1998-04-23 | Ag-Gene Australia Limited | Synthetic genes and genetic constructs comprising same I |
| US20020037281A1 (en) * | 2000-05-26 | 2002-03-28 | Davidson Beverly L. | Methods of transducing neural cells using lentivirus vectors |
| US20040219671A1 (en) * | 2002-02-20 | 2004-11-04 | Sirna Therapeutics, Inc. | RNA interference mediated treatment of parkinson disease using short interfering nucleic acid (siNA) |
| GB0130955D0 (en) * | 2001-12-24 | 2002-02-13 | Cancer Res Ventures | Expression system |
| US7820632B2 (en) * | 2002-02-14 | 2010-10-26 | City Of Hope | Methods for producing interfering RNA molecules in mammalian cells and therapeutic uses for such molecules |
| US20040023390A1 (en) * | 2002-08-05 | 2004-02-05 | Davidson Beverly L. | SiRNA-mediated gene silencing with viral vectors |
| AU2003295600A1 (en) * | 2002-11-14 | 2004-06-15 | Dharmacon, Inc. | Functional and hyperfunctional sirna |
| US7605249B2 (en) * | 2002-11-26 | 2009-10-20 | Medtronic, Inc. | Treatment of neurodegenerative disease through intracranial delivery of siRNA |
| WO2005004794A2 (en) * | 2003-06-09 | 2005-01-20 | Alnylam Pharmaceuticals Inc. | Method of treating neurodegenerative disease |
-
2005
- 2005-09-27 CA CA2580189A patent/CA2580189C/en not_active Expired - Fee Related
- 2005-09-27 DE DE602005015994T patent/DE602005015994D1/de active Active
- 2005-09-27 AT AT05816006T patent/ATE439440T1/de not_active IP Right Cessation
- 2005-09-27 EP EP05816006A patent/EP1799826B1/de active Active
- 2005-09-27 WO PCT/US2005/034516 patent/WO2006039253A2/en not_active Ceased
-
2007
- 2007-03-21 US US11/726,211 patent/US20070172462A1/en not_active Abandoned
-
2010
- 2010-01-25 US US12/693,101 patent/US8389487B2/en not_active Expired - Fee Related
Also Published As
| Publication number | Publication date |
|---|---|
| WO2006039253A3 (en) | 2006-11-23 |
| US20100286242A1 (en) | 2010-11-11 |
| EP1799826B1 (de) | 2009-08-12 |
| CA2580189A1 (en) | 2006-04-13 |
| US20070172462A1 (en) | 2007-07-26 |
| EP1799826A2 (de) | 2007-06-27 |
| DE602005015994D1 (de) | 2009-09-24 |
| WO2006039253A2 (en) | 2006-04-13 |
| US8389487B2 (en) | 2013-03-05 |
| CA2580189C (en) | 2013-05-21 |
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Legal Events
| Date | Code | Title | Description |
|---|---|---|---|
| RER | Ceased as to paragraph 5 lit. 3 law introducing patent treaties |