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WO2006078798A3 - RNA INTERFERENCE MEDIATED INHIBITION OF RETINOBLASTOMA (RB1) GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA) - Google Patents

RNA INTERFERENCE MEDIATED INHIBITION OF RETINOBLASTOMA (RB1) GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA) Download PDF

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Publication number
WO2006078798A3
WO2006078798A3 PCT/US2006/001863 US2006001863W WO2006078798A3 WO 2006078798 A3 WO2006078798 A3 WO 2006078798A3 US 2006001863 W US2006001863 W US 2006001863W WO 2006078798 A3 WO2006078798 A3 WO 2006078798A3
Authority
WO
WIPO (PCT)
Prior art keywords
nucleic acid
retinoblastoma
short interfering
sina
rna
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Ceased
Application number
PCT/US2006/001863
Other languages
French (fr)
Other versions
WO2006078798A2 (en
Inventor
Angela Christiano
James Mcswiggen
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Sirna Therapeutics Inc
Original Assignee
Sirna Therapeutics Inc
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Priority claimed from US11/058,582 external-priority patent/US20050260620A1/en
Application filed by Sirna Therapeutics Inc filed Critical Sirna Therapeutics Inc
Priority to EP06718871A priority Critical patent/EP1844147A2/en
Publication of WO2006078798A2 publication Critical patent/WO2006078798A2/en
Publication of WO2006078798A3 publication Critical patent/WO2006078798A3/en
Anticipated expiration legal-status Critical
Ceased legal-status Critical Current

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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
    • 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
    • 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]

Landscapes

  • Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Chemical & Material Sciences (AREA)
  • Biotechnology (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Molecular Biology (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Oncology (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Physics & Mathematics (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)

Abstract

This invention relates to compounds, compositions, and methods useful for modulating retinoblastoma (RBl) gene expression using short interfering nucleic acid (siNA) molecules. This invention also relates to compounds, compositions, and methods useful for modulating the expression and activity of other genes involved in pathways of retinoblastoma gene expression and/or activity by RNA interference (RNAi) using small nucleic acid molecules. In particular, the instant invention features small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), micro-RNA (miRNA), and short hairpin RNA (shRNA) molecules and methods used to modulate the expression of retinoblastoma genes. Such small nucleic acid molecules are useful, for example, for treating, preventing, inhibiting, or reducing hearing loss, deafness, tinnitus, motion and balance disorders, cancer and proliferative diseases and any other disease, condition, trait or indication that can respond to the level of retinoblastoma in a cell or tissue, alone or in combination with other treatments or therapies.
PCT/US2006/001863 2005-01-18 2006-01-18 RNA INTERFERENCE MEDIATED INHIBITION OF RETINOBLASTOMA (RB1) GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA) Ceased WO2006078798A2 (en)

Priority Applications (1)

Application Number Priority Date Filing Date Title
EP06718871A EP1844147A2 (en) 2005-01-18 2006-01-18 RNA INTERFERENCE MEDIATED INHIBITION OF RETINOBLASTOMA (RBI) GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA)

Applications Claiming Priority (10)

Application Number Priority Date Filing Date Title
US3968005A 2005-01-18 2005-01-18
US11/039,680 2005-01-18
US11/058,582 US20050260620A1 (en) 2001-05-18 2005-02-15 RNA interference mediated inhibition of retinolblastoma (RBI) gene expression using short interfering nucleic acid (siNA)
US11/058,582 2005-02-15
US67853105P 2005-05-06 2005-05-06
US60/678,531 2005-05-06
US70394605P 2005-07-29 2005-07-29
US60/703,946 2005-07-29
US73702405P 2005-11-15 2005-11-15
US60/737,024 2005-11-15

Publications (2)

Publication Number Publication Date
WO2006078798A2 WO2006078798A2 (en) 2006-07-27
WO2006078798A3 true WO2006078798A3 (en) 2007-01-25

Family

ID=36692864

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2006/001863 Ceased WO2006078798A2 (en) 2005-01-18 2006-01-18 RNA INTERFERENCE MEDIATED INHIBITION OF RETINOBLASTOMA (RB1) GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA)

Country Status (2)

Country Link
EP (1) EP1844147A2 (en)
WO (1) WO2006078798A2 (en)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2006125977A2 (en) * 2005-05-25 2006-11-30 The University Of York Hybrid interfering rna
EP3548005A4 (en) 2016-11-29 2020-06-17 Puretech Health LLC EXOSOME FOR THE OUTPUT OF THERAPEUTIC ACTIVE SUBSTANCES

Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2003070918A2 (en) * 2002-02-20 2003-08-28 Ribozyme Pharmaceuticals, Incorporated Rna interference by modified short interfering nucleic acid
WO2005028642A2 (en) * 2003-09-19 2005-03-31 Integrin Advanced Biosystems Ltd Method of culturing cells including the inhibition of the expression of a gene or genes involved in cell cycle control
WO2005042705A2 (en) * 2003-10-22 2005-05-12 The Trustees Of The University Of Pennsylvania Short interfering rna and micro-rna compounds and methods of designing, making and using the same
US20050260620A1 (en) * 2001-05-18 2005-11-24 Sirna Therapeutics, Inc. RNA interference mediated inhibition of retinolblastoma (RBI) gene expression using short interfering nucleic acid (siNA)

Patent Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20050260620A1 (en) * 2001-05-18 2005-11-24 Sirna Therapeutics, Inc. RNA interference mediated inhibition of retinolblastoma (RBI) gene expression using short interfering nucleic acid (siNA)
WO2003070918A2 (en) * 2002-02-20 2003-08-28 Ribozyme Pharmaceuticals, Incorporated Rna interference by modified short interfering nucleic acid
WO2005028642A2 (en) * 2003-09-19 2005-03-31 Integrin Advanced Biosystems Ltd Method of culturing cells including the inhibition of the expression of a gene or genes involved in cell cycle control
WO2005042705A2 (en) * 2003-10-22 2005-05-12 The Trustees Of The University Of Pennsylvania Short interfering rna and micro-rna compounds and methods of designing, making and using the same

Non-Patent Citations (5)

* Cited by examiner, † Cited by third party
Title
ALLERSON CHARLES R ET AL: "Fully 2'-modified oligonucleotide duplexes with improved in vitro potency and stability compared to unmodified small interfering RNA", JOURNAL OF MEDICINAL CHEMISTRY, vol. 48, no. 4, 24 February 2005 (2005-02-24), pages 901 - 904, XP002404763, ISSN: 0022-2623 *
CZAUDERNA FRANK ET AL: "Structural variations and stabilising modifications of synthetic siRNAs in mammalian cells.", NUCLEIC ACIDS RESEARCH, vol. 31, no. 11, 1 June 2003 (2003-06-01), pages 2705 - 2716, XP002270732, ISSN: 0305-1048 *
HUANG HAOJIE ET AL: "Androgens repress Bcl-2 expression via activation of the retinoblastoma (RB) protein in prostate cancer cells.", ONCOGENE, vol. 23, no. 12, 18 March 2004 (2004-03-18), pages 2161 - 2176, XP002404761, ISSN: 0950-9232 *
PRAKASH THAZHA P ET AL: "Positional effect of chemical modifications on short interference RNA activity in mammalian cells", JOURNAL OF MEDICINAL CHEMISTRY, vol. 48, no. 13, June 2005 (2005-06-01), pages 4247 - 4253, XP002404764, ISSN: 0022-2623 *
SEMIZAROV DIMITRI ET AL: "siRNA-mediated gene silencing: a global genome view", NUCLEIC ACIDS RESEARCH, vol. 32, no. 13, 2004, pages 3836 - 3845, XP002404760, ISSN: 0305-1048 *

Also Published As

Publication number Publication date
WO2006078798A2 (en) 2006-07-27
EP1844147A2 (en) 2007-10-17

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