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)
- Pharmacology & Pharmacy (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Medicinal Chemistry (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Microbiology (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (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 (43)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN1747747B (zh) * | 2003-01-06 | 2012-09-05 | 安吉奥开米公司 | 作为穿过血脑屏障的载体的抑酶肽及类似物 |
| US20090016959A1 (en) * | 2005-02-18 | 2009-01-15 | Richard Beliveau | Delivery of antibodies to the central nervous system |
| CN101160403B (zh) * | 2005-02-18 | 2014-08-13 | 安吉奥开米公司 | 转运化合物穿过血脑屏障的分子 |
| JP5436856B2 (ja) | 2005-07-15 | 2014-03-05 | アンジオケム インコーポレーティッド | 薬学的複合体における担体としてのアプロチニンポリペプチドの使用 |
| US20090010894A1 (en) * | 2005-12-23 | 2009-01-08 | The Parkinson's Institute | Methods and systems for identifying compounds that modulate alpha-synuclein aggregation |
| WO2007098611A1 (en) * | 2006-03-03 | 2007-09-07 | Queen's University At Kingston | 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 |
| RU2518240C2 (ru) * | 2008-04-18 | 2014-06-10 | Ангиочем Инк. | Композиция, на основе гидрофобных агентов и способ ее получения(варианты) |
| RU2531591C2 (ru) | 2008-10-15 | 2014-10-20 | Ангиокем Инк. | Конъюгаты этопозида и доксорубицина для доставки лекарственных средств |
| US8921314B2 (en) | 2008-10-15 | 2014-12-30 | Angiochem, Inc. | Conjugates of GLP-1 agonists and uses thereof |
| MX2011005963A (es) * | 2008-12-05 | 2011-09-01 | Angiochem Inc | Conjugados de neurotensina o analogos de neurotensina y sus usos. |
| CN102300987A (zh) | 2008-12-17 | 2011-12-28 | 安吉奥开米公司 | 膜型-1基质金属蛋白抑制剂及其用途 |
| AU2010239069B2 (en) | 2009-04-20 | 2015-05-14 | Angiochem Inc | Treatment of ovarian cancer using an anticancer agent conjugated to an Angiopep-2 analog |
| 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 |
| MX2012000016A (es) | 2009-07-02 | 2012-03-26 | Angiochem Inc | Conjugados de peptidos multimericos y sus usos. |
| MX2012004247A (es) * | 2009-10-06 | 2012-06-25 | Angiochem Inc | Composiciones y metodos para el transporte de agentes terapeuticos. |
| EP2380595A1 (de) | 2010-04-19 | 2011-10-26 | Nlife Therapeutics S.L. | Zusammensetzungen und Verfahren zur selektiven Abgabe von Oligonukleotidmolekülen an spezifische Neuronentypen |
| WO2012068405A2 (en) | 2010-11-17 | 2012-05-24 | Isis Pharmaceuticals, Inc. | Modulation of alpha synuclein expression |
| US20150037257A1 (en) * | 2011-06-16 | 2015-02-05 | Sally Mak | Compositions and methods for high-throughput screening in skin fibroblasts with an alpha-synuclein triplication |
| CA2856137A1 (en) * | 2011-11-22 | 2013-05-30 | The Children's Hospital Of Philadelphia | Virus vectors for highly efficient transgene delivery |
| PT3024498T (pt) | 2013-07-22 | 2020-03-06 | Childrens Hospital Philadelphia | Variante aav e composições, métodos e usos para transferência genética em células, órgãos e tecidos |
| 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 |
| US10980892B2 (en) | 2015-06-15 | 2021-04-20 | Angiochem Inc. | Methods for the treatment of leptomeningeal carcinomatosis |
| PT3313991T (pt) | 2015-06-23 | 2024-09-27 | Childrens Hospital Philadelphia | Fator ix modificado e composições, métodos e utilizações para transferência de genes para células, órgãos e tecidos |
| EP3538095A4 (de) | 2016-11-14 | 2020-06-17 | Berg LLC | Verfahren zur behandlung von morbus parkinson |
| CN112553210B (zh) | 2017-10-03 | 2024-09-24 | 普利维尔治疗公司 | 用于溶酶体障碍的基因疗法 |
| CA3078501A1 (en) | 2017-10-03 | 2019-04-11 | Prevail Therapeutics, Inc. | Gene therapies for lysosomal disorders |
| BR112020006633A2 (pt) | 2017-10-03 | 2020-10-06 | Prevail Therapeutics, Inc. | terapias genéticas para distúrbios lisossômicos |
| 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 |
| MA51634A (fr) | 2018-01-12 | 2020-11-18 | Roche Innovation Ct Copenhagen As | Oligonucléotides antisens d'alpha-synucléine et leurs utilisations |
| US12241065B2 (en) | 2018-01-12 | 2025-03-04 | Bristol-Myers Squibb Company | 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. |
| KR20220006527A (ko) | 2019-04-10 | 2022-01-17 | 프리베일 테라퓨틱스, 인크. | 리소좀 장애에 대한 유전자 요법 |
| US20250382611A1 (en) * | 2021-06-21 | 2025-12-18 | 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 |
| AU2024363678A1 (en) * | 2023-10-20 | 2026-05-07 | Spark Therapeutics, Inc. | Inhibitory nucleic acid targeting alpha synuclein expression |
| CN118512601B (zh) * | 2024-07-23 | 2024-12-10 | 深川生物科技(山东)股份公司 | 双链核酸-半乳糖胺-胆固醇化合物及其应用 |
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 |
| CA2410015A1 (en) * | 2000-05-26 | 2001-12-06 | Thomas W. Dubensky, Jr. | 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 |
| WO2003068797A1 (en) * | 2002-02-14 | 2003-08-21 | 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 |
| 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 |
| US7605249B2 (en) * | 2002-11-26 | 2009-10-20 | Medtronic, Inc. | Treatment of neurodegenerative disease through intracranial delivery of siRNA |
| CA2542232A1 (en) * | 2003-06-09 | 2005-01-20 | Alnylam Pharmaceuticals, Inc. | Method for treating neurodegenerative disease by inhibiting alpha-synuclein |
-
2005
- 2005-09-27 EP EP05816006A patent/EP1799826B1/de active Active
- 2005-09-27 DE DE602005015994T patent/DE602005015994D1/de active Active
- 2005-09-27 WO PCT/US2005/034516 patent/WO2006039253A2/en not_active Ceased
- 2005-09-27 AT AT05816006T patent/ATE439440T1/de not_active IP Right Cessation
- 2005-09-27 CA CA2580189A patent/CA2580189C/en not_active Expired - Fee Related
-
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 |
|---|---|
| DE602005015994D1 (de) | 2009-09-24 |
| US8389487B2 (en) | 2013-03-05 |
| EP1799826B1 (de) | 2009-08-12 |
| WO2006039253A2 (en) | 2006-04-13 |
| US20070172462A1 (en) | 2007-07-26 |
| CA2580189A1 (en) | 2006-04-13 |
| EP1799826A2 (de) | 2007-06-27 |
| WO2006039253A3 (en) | 2006-11-23 |
| US20100286242A1 (en) | 2010-11-11 |
| 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 |