ATE522626T1 - Verfahren und nukleotid-sequenzen, die bevorzugt die expression eines mutierten huntingtin-genes unterdrücken - Google Patents
Verfahren und nukleotid-sequenzen, die bevorzugt die expression eines mutierten huntingtin-genes unterdrückenInfo
- Publication number
- ATE522626T1 ATE522626T1 AT09003559T AT09003559T ATE522626T1 AT E522626 T1 ATE522626 T1 AT E522626T1 AT 09003559 T AT09003559 T AT 09003559T AT 09003559 T AT09003559 T AT 09003559T AT E522626 T1 ATE522626 T1 AT E522626T1
- Authority
- AT
- Austria
- Prior art keywords
- expression
- methods
- htt
- preferentially suppress
- mutated huntingtin
- Prior art date
Links
- 108090000623 proteins and genes Proteins 0.000 title abstract 5
- 230000014509 gene expression Effects 0.000 title abstract 4
- 238000000034 method Methods 0.000 title abstract 3
- 108091028043 Nucleic acid sequence Proteins 0.000 title abstract 2
- 125000003275 alpha amino acid group Chemical group 0.000 abstract 2
- 239000002773 nucleotide Substances 0.000 abstract 2
- 125000003729 nucleotide group Chemical group 0.000 abstract 2
- 102000054765 polymorphisms of proteins Human genes 0.000 abstract 2
- 102000004169 proteins and genes Human genes 0.000 abstract 2
- 208000023105 Huntington disease Diseases 0.000 abstract 1
- 108020004707 nucleic acids Proteins 0.000 abstract 1
- 102000039446 nucleic acids Human genes 0.000 abstract 1
- 150000007523 nucleic acids Chemical class 0.000 abstract 1
- 230000002018 overexpression Effects 0.000 abstract 1
- 238000012216 screening Methods 0.000 abstract 1
- 230000008685 targeting Effects 0.000 abstract 1
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
- C12N15/111—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
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Q—MEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
- C12Q1/00—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
- C12Q1/68—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving nucleic acids
- C12Q1/6876—Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes
- C12Q1/6883—Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes for diseases caused by alterations of genetic material
-
- 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
- C12N2310/50—Physical structure
- C12N2310/53—Physical structure partially self-complementary or closed
-
- 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
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Q—MEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
- C12Q2600/00—Oligonucleotides characterized by their use
- C12Q2600/156—Polymorphic or mutational markers
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Q—MEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
- C12Q2600/00—Oligonucleotides characterized by their use
- C12Q2600/158—Expression markers
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Chemical & Material Sciences (AREA)
- Biomedical Technology (AREA)
- Organic Chemistry (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- Molecular Biology (AREA)
- General Engineering & Computer Science (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biotechnology (AREA)
- Microbiology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Biochemistry (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- General Health & Medical Sciences (AREA)
- Plant Pathology (AREA)
- Analytical Chemistry (AREA)
- Immunology (AREA)
- Pathology (AREA)
- Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Peptides Or Proteins (AREA)
- Investigating Or Analysing Biological Materials (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
Applications Claiming Priority (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US69507805P | 2005-06-28 | 2005-06-28 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| ATE522626T1 true ATE522626T1 (de) | 2011-09-15 |
Family
ID=37461493
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| AT09003559T ATE522626T1 (de) | 2005-06-28 | 2006-06-28 | Verfahren und nukleotid-sequenzen, die bevorzugt die expression eines mutierten huntingtin-genes unterdrücken |
Country Status (4)
| Country | Link |
|---|---|
| US (1) | US20070161590A1 (de) |
| EP (4) | EP2062980B1 (de) |
| AT (1) | ATE522626T1 (de) |
| WO (1) | WO2007002904A2 (de) |
Families Citing this family (39)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US8680063B2 (en) | 2003-09-12 | 2014-03-25 | University Of Massachusetts | RNA interference for the treatment of gain-of-function disorders |
| DK2821085T3 (da) | 2003-09-12 | 2020-08-03 | Univ Massachusetts | Rna-interferens til behandling af "gain-of-function"-forstyrrelser |
| WO2008005562A2 (en) * | 2006-07-07 | 2008-01-10 | University Of Massachusetts | Rna silencing compositions and methods for the treatment of huntington's disease |
| US9133517B2 (en) * | 2005-06-28 | 2015-09-15 | Medtronics, Inc. | Methods and sequences to preferentially suppress expression of mutated huntingtin |
| EP1937066A4 (de) | 2005-08-18 | 2008-12-24 | Alnylam Pharmaceuticals Inc | Verfahren und zusammensetzungen für die behandlung von nervenkrankheiten |
| US9273356B2 (en) * | 2006-05-24 | 2016-03-01 | Medtronic, Inc. | Methods and kits for linking polymorphic sequences to expanded repeat mutations |
| WO2008143774A2 (en) * | 2007-05-01 | 2008-11-27 | University Of Massachusetts | Methods and compositions for locating snp heterozygosity for allele specific diagnosis and therapy |
| WO2009008727A2 (en) | 2007-07-12 | 2009-01-15 | Prosensa Technologies B.V. | Molecules for targeting compounds to various selected organs or tissues |
| CA2693742A1 (en) | 2007-07-12 | 2009-01-15 | Prosensa Technologies B.V. | Molecules for targeting compounds to various selected organs, tissues or tumor cells |
| ES2639852T3 (es) | 2007-10-26 | 2017-10-30 | Academisch Ziekenhuis Leiden | Medios y métodos para contrarrestar los trastornos musculares |
| USRE48468E1 (en) | 2007-10-26 | 2021-03-16 | Biomarin Technologies B.V. | Means and methods for counteracting muscle disorders |
| WO2009099326A1 (en) | 2008-02-08 | 2009-08-13 | Prosensa Holding Bv | Methods and means for treating dna repeat instability associated genetic disorders |
| CA2726866A1 (en) * | 2008-05-09 | 2009-11-12 | The University Of British Columbia | Methods and compositions for the treatment of huntington's disease |
| US8679750B2 (en) | 2008-05-09 | 2014-03-25 | The University Of British Columbia | Methods and compositions for the treatment of Huntington'S disease |
| EP2119783A1 (de) | 2008-05-14 | 2009-11-18 | Prosensa Technologies B.V. | Verfahren für effizientes Exon (44)-Skipping bei Duchenne-Muskeldystrophie und entsprechende Mittel |
| CN102282155B (zh) | 2008-12-02 | 2017-06-09 | 日本波涛生命科学公司 | 磷原子修饰的核酸的合成方法 |
| CA2756670A1 (en) * | 2009-03-26 | 2010-09-30 | The Regents Of The University Of California | Mesenchymal stem cells producing inhibitory rna for disease modification |
| EP2421971B1 (de) | 2009-04-24 | 2016-07-06 | BioMarin Technologies B.V. | Oligonukleotid mit einem inosin zur behandlung von dmd |
| RU2612521C2 (ru) | 2009-07-06 | 2017-03-09 | Онтории, Инк. | Новые пролекарства нуклеиновых кислот и способы их применения |
| CA2789038A1 (en) | 2010-02-08 | 2011-08-11 | Isis Pharmaceuticals, Inc. | Selective reduction of allelic variants |
| US8957040B2 (en) | 2010-02-08 | 2015-02-17 | Isis Pharmaceuticals, Inc. | Selective reduction of allelic variants |
| JP5868324B2 (ja) | 2010-09-24 | 2016-02-24 | 株式会社Wave Life Sciences Japan | 不斉補助基 |
| CN103796657B (zh) | 2011-07-19 | 2017-07-11 | 波涛生命科学有限公司 | 合成官能化核酸的方法 |
| US10202599B2 (en) | 2011-08-11 | 2019-02-12 | Ionis Pharmaceuticals, Inc. | Selective antisense compounds and uses thereof |
| NZ627896A (en) | 2012-01-27 | 2016-11-25 | Biomarin Technologies B V | Rna modulating oligonucleotides with improved characteristics for the treatment of duchenne and becker muscular dystrophy |
| CA2879066C (en) | 2012-07-13 | 2019-08-13 | Shin Nippon Biomedical Laboratories, Ltd. | Chiral nucleic acid adjuvant |
| KR102213609B1 (ko) | 2012-07-13 | 2021-02-08 | 웨이브 라이프 사이언시스 리미티드 | 키랄 제어 |
| PL2872485T3 (pl) | 2012-07-13 | 2021-05-31 | Wave Life Sciences Ltd. | Asymetryczna grupa pomocnicza |
| EP3459549B1 (de) | 2012-10-12 | 2022-04-06 | Ionis Pharmaceuticals, Inc. | Selektive antisense-verbindungen und verwendungen davon |
| JPWO2015108047A1 (ja) | 2014-01-15 | 2017-03-23 | 株式会社新日本科学 | 免疫誘導活性を有するキラル核酸アジュバンド及び免疫誘導活性剤 |
| US10322173B2 (en) | 2014-01-15 | 2019-06-18 | Shin Nippon Biomedical Laboratories, Ltd. | Chiral nucleic acid adjuvant having anti-allergic activity, and anti-allergic agent |
| JPWO2015108048A1 (ja) | 2014-01-15 | 2017-03-23 | 株式会社新日本科学 | 抗腫瘍作用を有するキラル核酸アジュバンド及び抗腫瘍剤 |
| BR112016016400A2 (pt) | 2014-01-16 | 2017-10-03 | Wave Life Sciences Ltd | Composições de oligonucleotídeos quiralmente controlados, seu uso, sua composição farmacêutica, e métodos |
| IL320434A (en) | 2015-07-22 | 2025-06-01 | Wave Life Sciences Ltd | Oligonucleotide preparations and methods |
| WO2017141157A1 (en) * | 2016-02-15 | 2017-08-24 | Istituti Clinici Scientifici Maugeri Spa Sb | Method of allele specific silencing for the treatment of autosomal dominant catecholaminergic polymorphic ventricular tachycardia (cpvt) |
| EP3452596A4 (de) | 2016-05-04 | 2020-03-18 | Wave Life Sciences Ltd. | Oligonukleotidzusammensetzungen und verfahren dafür |
| CN110177887B (zh) | 2016-11-10 | 2024-10-29 | 达丽斯生物医学公司 | 用于扩增和检测沙眼衣原体的多核苷酸 |
| CN106884047B (zh) * | 2017-02-15 | 2021-03-23 | 济南大学 | 基于核酸适配体检测miRNA-155的方法 |
| US11047007B1 (en) | 2020-03-23 | 2021-06-29 | Talis Biomedical Corporation | Polynucleotides for amplification and detection of SARS-CoV-2 |
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| US20050202075A1 (en) * | 2004-03-12 | 2005-09-15 | Pardridge William M. | Delivery of genes encoding short hairpin RNA using receptor-specific nanocontainers |
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-
2006
- 2006-06-28 WO PCT/US2006/025638 patent/WO2007002904A2/en not_active Ceased
- 2006-06-28 US US11/478,110 patent/US20070161590A1/en not_active Abandoned
- 2006-06-28 EP EP09003559A patent/EP2062980B1/de not_active Not-in-force
- 2006-06-28 EP EP10012595.4A patent/EP2322657B1/de not_active Not-in-force
- 2006-06-28 EP EP06785990A patent/EP1896586A2/de not_active Withdrawn
- 2006-06-28 EP EP10012594A patent/EP2316967A1/de not_active Withdrawn
- 2006-06-28 AT AT09003559T patent/ATE522626T1/de not_active IP Right Cessation
Also Published As
| Publication number | Publication date |
|---|---|
| US20070161590A1 (en) | 2007-07-12 |
| EP2062980A3 (de) | 2009-08-26 |
| WO2007002904A2 (en) | 2007-01-04 |
| EP2316967A1 (de) | 2011-05-04 |
| EP2322657A1 (de) | 2011-05-18 |
| EP2062980B1 (de) | 2011-08-31 |
| WO2007002904A3 (en) | 2007-08-02 |
| EP1896586A2 (de) | 2008-03-12 |
| EP2062980A2 (de) | 2009-05-27 |
| EP2322657B1 (de) | 2014-11-05 |
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