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
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)
- Organic Chemistry (AREA)
- Biomedical Technology (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Molecular Biology (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biophysics (AREA)
- Microbiology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Biochemistry (AREA)
- General Health & Medical Sciences (AREA)
- Physics & Mathematics (AREA)
- Analytical Chemistry (AREA)
- Plant Pathology (AREA)
- Pathology (AREA)
- Immunology (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
- Peptides Or Proteins (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Investigating Or Analysing Biological Materials (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) | EP2322657B1 (de) |
AT (1) | ATE522626T1 (de) |
WO (1) | WO2007002904A2 (de) |
Families Citing this family (37)
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US8680063B2 (en) | 2003-09-12 | 2014-03-25 | University Of Massachusetts | RNA interference for the treatment of gain-of-function disorders |
EP2821085B1 (de) * | 2003-09-12 | 2020-04-29 | University of Massachusetts | Rna-interferenz zur behandlung von funktionszunahmestörungen |
US9133517B2 (en) * | 2005-06-28 | 2015-09-15 | Medtronics, Inc. | Methods and sequences to preferentially suppress expression of mutated huntingtin |
US20070161591A1 (en) * | 2005-08-18 | 2007-07-12 | University Of Massachusetts | Methods and compositions for treating neurological disease |
US9273356B2 (en) * | 2006-05-24 | 2016-03-01 | Medtronic, Inc. | Methods and kits for linking polymorphic sequences to expanded repeat mutations |
CA2662704A1 (en) * | 2006-07-07 | 2008-01-10 | University Of Massachusetts | Rna silencing compositions and methods for the treatment of huntington's disease |
WO2008143774A2 (en) * | 2007-05-01 | 2008-11-27 | University Of Massachusetts | Methods and compositions for locating snp heterozygosity for allele specific diagnosis and therapy |
EP2167135A2 (de) | 2007-07-12 | 2010-03-31 | Prosensa Technologies B.V. | Moleküle für targeting von verbindungen auf verschiedene ausgewählte organe, gewebe oder tumorzellen |
CN103212085A (zh) | 2007-07-12 | 2013-07-24 | 普罗森那技术公司 | 用于使化合物靶向多种选定器官或组织的分子 |
CN101896186A (zh) | 2007-10-26 | 2010-11-24 | 莱顿教学医院 | 对抗肌肉病症的方式和方法 |
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 |
AU2009244013B2 (en) * | 2008-05-09 | 2015-06-25 | 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 |
CA2744987C (en) | 2008-12-02 | 2018-01-16 | Chiralgen, Ltd. | Method for the synthesis of phosphorus atom modified nucleic acids |
EP2411505A4 (de) * | 2009-03-26 | 2013-01-30 | Univ California | Mesenchymale stammzellen zur produktion hemmender rna zur krankheitsmodifikation |
ES2593836T3 (es) | 2009-04-24 | 2016-12-13 | Biomarin Technologies B.V. | Oligonucleótido que comprende una inosina para tratar la DMD |
AU2010270714B2 (en) | 2009-07-06 | 2015-08-13 | Wave Life Sciences Ltd. | Novel nucleic acid prodrugs and methods use thereof |
JP6018506B2 (ja) * | 2010-02-08 | 2016-11-02 | アイオーニス ファーマシューティカルズ, インコーポレーテッドIonis Pharmaceuticals,Inc. | 対立遺伝子多様体の選択的低減 |
WO2011097643A1 (en) * | 2010-02-08 | 2011-08-11 | Isis Pharmaceuticals, Inc. | Selective reduction of allelic variants |
EP2620428B1 (de) | 2010-09-24 | 2019-05-22 | Wave Life Sciences Ltd. | Asymmetrische hilfsgruppe |
SG10201700554VA (en) | 2011-07-19 | 2017-03-30 | Wave Life Sciences Pte Ltd | Methods for the synthesis of functionalized nucleic acids |
EP2742056B2 (de) | 2011-08-11 | 2020-06-10 | Ionis Pharmaceuticals, Inc. | Selektive antisense-verbindungen und ihre verwendungen |
EP4043039A1 (de) | 2012-01-27 | 2022-08-17 | BioMarin Technologies B.V. | Rna-modulierende oligonukleotide mit verbesserten eigenschaften zur behandlung der duchenne- und becker-muskeldystrophie |
WO2014012081A2 (en) | 2012-07-13 | 2014-01-16 | Ontorii, Inc. | Chiral control |
SG11201500239VA (en) | 2012-07-13 | 2015-03-30 | Wave Life Sciences Japan | Asymmetric auxiliary group |
BR112015000723A2 (pt) | 2012-07-13 | 2017-06-27 | Shin Nippon Biomedical Laboratories Ltd | adjuvante de ácido nucléico quiral |
CA2887884A1 (en) | 2012-10-12 | 2014-04-17 | Isis Pharmaceuticals, Inc. | Selective antisense compounds and uses thereof |
US10149905B2 (en) | 2014-01-15 | 2018-12-11 | Shin Nippon Biomedical Laboratories, Ltd. | Chiral nucleic acid adjuvant having antitumor effect and antitumor agent |
EP3095461A4 (de) | 2014-01-15 | 2017-08-23 | Shin Nippon Biomedical Laboratories, Ltd. | Chirales nukleinsäure-adjuvans mit immunitätsinduktionswirkung und immunitätsinduktionsaktivator |
JPWO2015108046A1 (ja) | 2014-01-15 | 2017-03-23 | 株式会社新日本科学 | 抗アレルギー作用を有するキラル核酸アジュバンド及び抗アレルギー剤 |
MX2016009290A (es) | 2014-01-16 | 2017-02-28 | Wave Life Sciences Ltd | Diseño quiral. |
MA43072A (fr) | 2015-07-22 | 2018-05-30 | Wave Life Sciences Ltd | Compositions d'oligonucléotides et procédés associés |
US11591602B2 (en) * | 2016-02-15 | 2023-02-28 | Istituti Clinici Scientifici Maugeri Spa Sb | Method of allele specific silencing for the treatment of autosomal dominant Catecholaminergic Polymorphic Ventricular Tachycardia (CPVT) |
MA45270A (fr) | 2016-05-04 | 2017-11-09 | Wave Life Sciences Ltd | Compositions d'oligonucléotides et procédés associés |
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 |
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EP2821085B1 (de) * | 2003-09-12 | 2020-04-29 | University of Massachusetts | Rna-interferenz zur behandlung von funktionszunahmestörungen |
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-
2006
- 2006-06-28 WO PCT/US2006/025638 patent/WO2007002904A2/en active Application Filing
- 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 EP EP09003559A patent/EP2062980B1/de not_active Not-in-force
- 2006-06-28 AT AT09003559T patent/ATE522626T1/de not_active IP Right Cessation
- 2006-06-28 US US11/478,110 patent/US20070161590A1/en not_active Abandoned
Also Published As
Publication number | Publication date |
---|---|
EP2322657A1 (de) | 2011-05-18 |
EP2062980A2 (de) | 2009-05-27 |
EP1896586A2 (de) | 2008-03-12 |
EP2316967A1 (de) | 2011-05-04 |
EP2322657B1 (de) | 2014-11-05 |
EP2062980B1 (de) | 2011-08-31 |
WO2007002904A3 (en) | 2007-08-02 |
US20070161590A1 (en) | 2007-07-12 |
EP2062980A3 (de) | 2009-08-26 |
WO2007002904A2 (en) | 2007-01-04 |
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