WO2003070972A3 - RNA INTERFERENCE MEDIATED INHIBITION OF CHROMOSOME TRANSLOCATION GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA) - Google Patents
RNA INTERFERENCE MEDIATED INHIBITION OF CHROMOSOME TRANSLOCATION GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA) Download PDFInfo
- Publication number
- WO2003070972A3 WO2003070972A3 PCT/US2003/005234 US0305234W WO03070972A3 WO 2003070972 A3 WO2003070972 A3 WO 2003070972A3 US 0305234 W US0305234 W US 0305234W WO 03070972 A3 WO03070972 A3 WO 03070972A3
- Authority
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- WIPO (PCT)
- Prior art keywords
- gene expression
- nucleic acid
- rna
- short interfering
- sina
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- 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
- C12N15/1135—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 oncogenes or tumor suppressor genes
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- 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
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/14—Type of nucleic acid interfering N.A.
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/31—Chemical structure of the backbone
- C12N2310/315—Phosphorothioates
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- 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
- C12N2310/30—Chemical structure
- C12N2310/31—Chemical structure of the backbone
- C12N2310/317—Chemical structure of the backbone with an inverted bond, e.g. a cap structure
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- 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
- C12N2310/30—Chemical structure
- C12N2310/31—Chemical structure of the backbone
- C12N2310/318—Chemical structure of the backbone where the PO2 is completely replaced, e.g. MMI or formacetal
-
- 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
- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
- C12N2310/321—2'-O-R Modification
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
- C12N2310/322—2'-R Modification
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- 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
- C12N2310/30—Chemical structure
- C12N2310/33—Chemical structure of the base
- C12N2310/332—Abasic residue
-
- 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
- C12N2310/30—Chemical structure
- C12N2310/34—Spatial arrangement of the modifications
- C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
-
- 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
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- Health & Medical Sciences (AREA)
- Genetics & Genomics (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Biomedical Technology (AREA)
- General Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
- Organic Chemistry (AREA)
- Chemical & Material Sciences (AREA)
- Biotechnology (AREA)
- Molecular Biology (AREA)
- Zoology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Oncology (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Biochemistry (AREA)
- General Health & Medical Sciences (AREA)
- Physics & Mathematics (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
Abstract
Priority Applications (5)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
EP03716110A EP1476459A4 (en) | 2002-02-20 | 2003-02-20 | RNA INTERFERENCE MEDIATED INHIBITION OF CHROMOSOME TRANSLOCATION GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA) |
AU2003219833A AU2003219833A1 (en) | 2002-02-20 | 2003-02-20 | RNA INTERFERENCE MEDIATED INHIBITION OF CHROMOSOME TRANSLOCATION GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA) |
US10/923,522 US20050159381A1 (en) | 2001-05-18 | 2004-08-20 | RNA interference mediated inhibition of chromosome translocation gene expression using short interfering nucleic acid (siNA) |
US12/205,558 US20090093439A1 (en) | 2002-02-20 | 2008-09-05 | RNA INTERFERENCE MEDIATED INHIBITION OF CHROMOSOME TRANSLOCATION GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA) |
US12/717,511 US20100227911A1 (en) | 2002-02-20 | 2010-03-04 | RNA INTERFERENCE MEDIATED INHIBITION OF CHROMOSOME TRANSLOCATION GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA) |
Applications Claiming Priority (18)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US35858002P | 2002-02-20 | 2002-02-20 | |
US60/358,580 | 2002-02-20 | ||
US36312402P | 2002-03-11 | 2002-03-11 | |
US60/363,124 | 2002-03-11 | ||
US38678202P | 2002-06-06 | 2002-06-06 | |
US60/386,782 | 2002-06-06 | ||
US40403902P | 2002-08-15 | 2002-08-15 | |
US60/404,039 | 2002-08-15 | ||
US40678402P | 2002-08-29 | 2002-08-29 | |
US60/406,784 | 2002-08-29 | ||
US40837802P | 2002-09-05 | 2002-09-05 | |
US60/408,378 | 2002-09-05 | ||
US40929302P | 2002-09-09 | 2002-09-09 | |
US60/409,293 | 2002-09-09 | ||
US43992203P | 2003-01-14 | 2003-01-14 | |
US60/439,922 | 2003-01-14 | ||
US44012903P | 2003-01-15 | 2003-01-15 | |
US60/440,129 | 2003-01-15 |
Related Child Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
US10/923,522 Continuation-In-Part US20050159381A1 (en) | 2001-05-18 | 2004-08-20 | RNA interference mediated inhibition of chromosome translocation gene expression using short interfering nucleic acid (siNA) |
Publications (3)
Publication Number | Publication Date |
---|---|
WO2003070972A2 WO2003070972A2 (en) | 2003-08-28 |
WO2003070972A9 WO2003070972A9 (en) | 2004-03-04 |
WO2003070972A3 true WO2003070972A3 (en) | 2004-06-03 |
Family
ID=27761736
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
PCT/US2003/005234 WO2003070972A2 (en) | 2001-05-18 | 2003-02-20 | RNA INTERFERENCE MEDIATED INHIBITION OF CHROMOSOME TRANSLOCATION GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA) |
Country Status (3)
Country | Link |
---|---|
EP (1) | EP1476459A4 (en) |
AU (1) | AU2003219833A1 (en) |
WO (1) | WO2003070972A2 (en) |
Families Citing this family (19)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
DE19956568A1 (en) | 1999-01-30 | 2000-08-17 | Roland Kreutzer | Method and medicament for inhibiting the expression of a given gene |
DE10100586C1 (en) | 2001-01-09 | 2002-04-11 | Ribopharma Ag | Inhibiting gene expression in cells, useful for e.g. treating tumors, by introducing double-stranded complementary oligoRNA having unpaired terminal bases |
US7829693B2 (en) | 1999-11-24 | 2010-11-09 | Alnylam Pharmaceuticals, Inc. | Compositions and methods for inhibiting expression of a target gene |
US8546143B2 (en) | 2001-01-09 | 2013-10-01 | Alnylam Pharmaceuticals, Inc. | Compositions and methods for inhibiting expression of a target gene |
US7767802B2 (en) | 2001-01-09 | 2010-08-03 | Alnylam Pharmaceuticals, Inc. | Compositions and methods for inhibiting expression of anti-apoptotic genes |
US7423142B2 (en) | 2001-01-09 | 2008-09-09 | Alnylam Pharmaceuticals, Inc. | Compositions and methods for inhibiting expression of anti-apoptotic genes |
DE10163098B4 (en) | 2001-10-12 | 2005-06-02 | Alnylam Europe Ag | Method for inhibiting the replication of viruses |
US7745418B2 (en) | 2001-10-12 | 2010-06-29 | Alnylam Pharmaceuticals, Inc. | Compositions and methods for inhibiting viral replication |
DE10202419A1 (en) | 2002-01-22 | 2003-08-07 | Ribopharma Ag | Method of inhibiting expression of a target gene resulting from chromosome aberration |
US9181551B2 (en) | 2002-02-20 | 2015-11-10 | Sirna Therapeutics, Inc. | RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (siNA) |
US9657294B2 (en) | 2002-02-20 | 2017-05-23 | Sirna Therapeutics, Inc. | RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (siNA) |
GB0216929D0 (en) * | 2002-07-20 | 2002-08-28 | Milner Anne J | Silencing of gene expression |
JP2008521401A (en) | 2004-11-24 | 2008-06-26 | アルナイラム ファーマシューティカルズ インコーポレイテッド | RNAi regulation of BCR-ABL fusion gene and method of use thereof |
US20110229552A1 (en) * | 2006-01-25 | 2011-09-22 | Erno Vreugdenhil | NOVEL mRNA SPLICE VARIANT OF THE DOUBLECORTIN-LIKE KINASE GENE AND ITS USE IN DIAGNOSIS AND THERAPY OF CANCERS OF NEUROECTODERMAL ORIGIN |
WO2008109376A1 (en) * | 2007-03-02 | 2008-09-12 | Mdrna, Inc. | Nucleic acid compounds for inhibiting bcr-abl gene expression and uses thereof |
US8143232B2 (en) * | 2009-03-03 | 2012-03-27 | The Regents Of The University Of Michigan | Gene fusion targeted therapy |
JP6106085B2 (en) | 2010-08-24 | 2017-03-29 | サーナ・セラピューティクス・インコーポレイテッドSirna Therapeutics,Inc. | Single-stranded RNAi agent containing an internal non-nucleic acid spacer |
DK2632472T3 (en) | 2010-10-29 | 2018-03-19 | Sirna Therapeutics Inc | RNA INTERFERENCE-MEDIATED INHIBITION OF GENE EXPRESSION USING SHORT INTERFERRING NUCLEIC ACIDS (SINA) |
RU2720475C2 (en) * | 2018-09-11 | 2020-04-30 | Федеральное государственное бюджетное образовательное учреждение высшего образования "Московский государственный университет имени М.В. Ломоносова" (МГУ) | Test system for searching for drugs which reduce the risk of secondary leukemia, a method for preparing and using it |
Citations (8)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
WO1994001550A1 (en) * | 1992-07-02 | 1994-01-20 | Hybridon, Inc. | Self-stabilized oligonucleotides as therapeutic agents |
WO1999032619A1 (en) * | 1997-12-23 | 1999-07-01 | The Carnegie Institution Of Washington | Genetic inhibition by double-stranded rna |
WO1999049029A1 (en) * | 1998-03-20 | 1999-09-30 | Benitec Australia Ltd | Control of gene expression |
WO2000001846A2 (en) * | 1998-07-03 | 2000-01-13 | Devgen N.V. | Characterisation of gene function using double stranded rna inhibition |
US6040181A (en) * | 1988-12-22 | 2000-03-21 | University Of Pennsylvania | Regulation of bcl-2 gene expression |
WO2000044914A1 (en) * | 1999-01-28 | 2000-08-03 | Medical College Of Georgia Research Institute, Inc. | Composition and method for in vivo and in vitro attenuation of gene expression using double stranded rna |
CA2359180A1 (en) * | 1999-01-30 | 2000-08-03 | Roland Kreutzer | Method and medicament for inhibiting the expression of a given gene |
WO2001036646A1 (en) * | 1999-11-19 | 2001-05-25 | Cancer Research Ventures Limited | Inhibiting gene expression with dsrna |
Family Cites Families (3)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
CA2323069A1 (en) * | 1998-03-12 | 1999-09-16 | Taisho Pharmaceutical Co., Ltd. | Nucleic acid enzyme showing allosteric rna-cleaving activity on target rna |
US6784291B2 (en) * | 2000-05-04 | 2004-08-31 | Avi Biopharma, Inc. | Splice-region antisense composition and method |
AU2003237686A1 (en) * | 2002-05-24 | 2003-12-12 | Max-Planck Gesellschaft Zur Forderung Der Wissenschaften E.V. | Rna interference mediating small rna molecules |
-
2003
- 2003-02-20 WO PCT/US2003/005234 patent/WO2003070972A2/en not_active Application Discontinuation
- 2003-02-20 AU AU2003219833A patent/AU2003219833A1/en not_active Abandoned
- 2003-02-20 EP EP03716110A patent/EP1476459A4/en not_active Withdrawn
Patent Citations (9)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
US6040181A (en) * | 1988-12-22 | 2000-03-21 | University Of Pennsylvania | Regulation of bcl-2 gene expression |
WO1994001550A1 (en) * | 1992-07-02 | 1994-01-20 | Hybridon, Inc. | Self-stabilized oligonucleotides as therapeutic agents |
WO1999032619A1 (en) * | 1997-12-23 | 1999-07-01 | The Carnegie Institution Of Washington | Genetic inhibition by double-stranded rna |
US6506559B1 (en) * | 1997-12-23 | 2003-01-14 | Carnegie Institute Of Washington | Genetic inhibition by double-stranded RNA |
WO1999049029A1 (en) * | 1998-03-20 | 1999-09-30 | Benitec Australia Ltd | Control of gene expression |
WO2000001846A2 (en) * | 1998-07-03 | 2000-01-13 | Devgen N.V. | Characterisation of gene function using double stranded rna inhibition |
WO2000044914A1 (en) * | 1999-01-28 | 2000-08-03 | Medical College Of Georgia Research Institute, Inc. | Composition and method for in vivo and in vitro attenuation of gene expression using double stranded rna |
CA2359180A1 (en) * | 1999-01-30 | 2000-08-03 | Roland Kreutzer | Method and medicament for inhibiting the expression of a given gene |
WO2001036646A1 (en) * | 1999-11-19 | 2001-05-25 | Cancer Research Ventures Limited | Inhibiting gene expression with dsrna |
Non-Patent Citations (5)
Title |
---|
KUWABARA T. ET AL.: "Activities of tRNA-embedded dimeric minizymes", NUCLEIC ACIDS SYMPOSIUM SERIES, vol. 37, January 1997 (1997-01-01), pages 307 - 308, XP002129252 * |
SCHERR M. ET AL.: "Specific inhibition of bcr-abl gene expression by small interfering RNA", BLOOD, vol. 101, no. 4, 15 February 2003 (2003-02-15), pages 1566 - 1569, XP002975208 * |
See also references of EP1476459A4 * |
WILDA M. ET AL.: "Killing of leukemic cells with a BCR/ABL fusion gene by RNA interference (RNAi)", ONCOGENE, vol. 21, June 2002 (2002-06-01), pages 5716 - 5724, XP009009806 * |
ZHOU J-M. ET AL.: "Probing of the secondary structure of maxizymes", NUCLEIC ACIDS SYMPOSIUM SERIES, no. 42, 1999, pages 219 - 220, XP002975209 * |
Also Published As
Publication number | Publication date |
---|---|
WO2003070972A9 (en) | 2004-03-04 |
EP1476459A4 (en) | 2005-05-25 |
AU2003219833A1 (en) | 2003-09-09 |
WO2003070972A2 (en) | 2003-08-28 |
AU2003219833A8 (en) | 2003-09-09 |
EP1476459A2 (en) | 2004-11-17 |
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