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WO2005117991A3 - Compositions and methods for enhancing delivery of nucleic acids into cells and for modifying expression of target genes in cells - Google Patents

Compositions and methods for enhancing delivery of nucleic acids into cells and for modifying expression of target genes in cells Download PDF

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Publication number
WO2005117991A3
WO2005117991A3 PCT/US2005/015574 US2005015574W WO2005117991A3 WO 2005117991 A3 WO2005117991 A3 WO 2005117991A3 US 2005015574 W US2005015574 W US 2005015574W WO 2005117991 A3 WO2005117991 A3 WO 2005117991A3
Authority
WO
WIPO (PCT)
Prior art keywords
cells
nucleic acids
methods
target genes
compositions
Prior art date
Application number
PCT/US2005/015574
Other languages
French (fr)
Other versions
WO2005117991A2 (en
Inventor
Lishan Chen
Kunyuan Cui
Yuching Chen
Sasha J Mayer
Original Assignee
Nastech Pharm Co
Lishan Chen
Kunyuan Cui
Yuching Chen
Sasha J Mayer
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
Application filed by Nastech Pharm Co, Lishan Chen, Kunyuan Cui, Yuching Chen, Sasha J Mayer filed Critical Nastech Pharm Co
Priority to MXPA06012605A priority Critical patent/MXPA06012605A/en
Priority to JP2007511576A priority patent/JP2007536253A/en
Priority to EP05804803A priority patent/EP1750775A2/en
Priority to CA002565685A priority patent/CA2565685A1/en
Publication of WO2005117991A2 publication Critical patent/WO2005117991A2/en
Publication of WO2005117991A3 publication Critical patent/WO2005117991A3/en

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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
    • C12N15/1136—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 growth factors, growth regulators, cytokines, lymphokines or hormones
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P29/00—Non-central analgesic, antipyretic or antiinflammatory agents, e.g. antirheumatic agents; Non-steroidal antiinflammatory drugs [NSAID]
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering N.A.
    • 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

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  • Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Veterinary Medicine (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pain & Pain Management (AREA)
  • Medicinal Chemistry (AREA)
  • Rheumatology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • General Chemical & Material Sciences (AREA)
  • Public Health (AREA)
  • Endocrinology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)

Abstract

Polynucleotide delivery-enhancing polypeptides are admixed or complexed with, or conjugated to, nucleic acids for enhancing delivery the nucleic acids into cells. The transported nucleic acids are active in target cells as small inhibitory nucleic acids (siNAs) that modulate expression of target genes, mediated at least in part by RNA interference (RNAi). The siNA/polypeptide compositions and methods of the invention provide effective tools to modulate gene expression and alter phenotype in mammalian cells, including by altering phenotype in a manner that eliminates disease symptoms or alters disease potential in targeted cells or subject individuals to which the siNA/polypeptide compositions are administered.
PCT/US2005/015574 2004-05-04 2005-05-04 Compositions and methods for enhancing delivery of nucleic acids into cells and for modifying expression of target genes in cells WO2005117991A2 (en)

Priority Applications (4)

Application Number Priority Date Filing Date Title
MXPA06012605A MXPA06012605A (en) 2004-05-04 2005-05-04 Compositions and methods for enhancing delivery of nucleic acids into cells and for modifying expression of target genes in cells.
JP2007511576A JP2007536253A (en) 2004-05-04 2005-05-04 Compositions and methods for enhancing delivery of nucleic acids into cells and modifying expression of target genes in cells
EP05804803A EP1750775A2 (en) 2004-05-04 2005-05-04 Compositions and methods for enhancing delivery of nucleic acids into cells and for modifying expression of target genes in cells
CA002565685A CA2565685A1 (en) 2004-05-04 2005-05-04 Compositions and methods for enhancing delivery of nucleic acids into cells and for modifying expression of target genes in cells

Applications Claiming Priority (12)

Application Number Priority Date Filing Date Title
US56802704P 2004-05-04 2004-05-04
US60/568,027 2004-05-04
US57051304P 2004-05-12 2004-05-12
US57051204P 2004-05-12 2004-05-12
US60/570,513 2004-05-12
US60/570,512 2004-05-12
US61341604P 2004-09-27 2004-09-27
US60/613,416 2004-09-27
US65657205P 2005-02-25 2005-02-25
US60/656,572 2005-02-25
US66783305P 2005-04-01 2005-04-01
US60/667,833 2005-04-01

Publications (2)

Publication Number Publication Date
WO2005117991A2 WO2005117991A2 (en) 2005-12-15
WO2005117991A3 true WO2005117991A3 (en) 2007-01-18

Family

ID=35311390

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2005/015574 WO2005117991A2 (en) 2004-05-04 2005-05-04 Compositions and methods for enhancing delivery of nucleic acids into cells and for modifying expression of target genes in cells

Country Status (5)

Country Link
EP (1) EP1750775A2 (en)
JP (1) JP2007536253A (en)
CA (1) CA2565685A1 (en)
MX (1) MXPA06012605A (en)
WO (1) WO2005117991A2 (en)

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US9228186B2 (en) 2002-11-14 2016-01-05 Thermo Fisher Scientific Inc. Methods and compositions for selecting siRNA of improved functionality

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US9719094B2 (en) 2002-11-14 2017-08-01 Thermo Fisher Scientific Inc. RNAi targeting SEC61G
US7951935B2 (en) 2002-11-14 2011-05-31 Dharmacon, Inc. siRNA targeting v-myc myelocytomatosis viral oncogene homolog (MYC)
WO2006006948A2 (en) 2002-11-14 2006-01-19 Dharmacon, Inc. METHODS AND COMPOSITIONS FOR SELECTING siRNA OF IMPROVED FUNCTIONALITY
US9839649B2 (en) 2002-11-14 2017-12-12 Thermo Fisher Scientific Inc. Methods and compositions for selecting siRNA of improved functionality
US9879266B2 (en) 2002-11-14 2018-01-30 Thermo Fisher Scientific Inc. Methods and compositions for selecting siRNA of improved functionality
US7612196B2 (en) 2002-11-14 2009-11-03 Dharmacon, Inc. siRNA targeting cyclin-dependent kinase inhibitor 1B (p27, Kip1) (CDKN1B)
US7691998B2 (en) 2002-11-14 2010-04-06 Dharmacon, Inc. siRNA targeting nucleoporin 62kDa (Nup62)
US8198427B1 (en) 2002-11-14 2012-06-12 Dharmacon, Inc. SiRNA targeting catenin, beta-1 (CTNNB1)
US7592442B2 (en) 2002-11-14 2009-09-22 Dharmacon, Inc. siRNA targeting ribonucleotide reductase M2 polypeptide (RRM2 or RNR-R2)
US7635770B2 (en) 2002-11-14 2009-12-22 Dharmacon, Inc. siRNA targeting protein kinase N-3 (PKN-3)
US10011836B2 (en) 2002-11-14 2018-07-03 Thermo Fisher Scientific Inc. Methods and compositions for selecting siRNA of improved functionality
US7977471B2 (en) 2002-11-14 2011-07-12 Dharmacon, Inc. siRNA targeting TNFα
EP2305813A3 (en) 2002-11-14 2012-03-28 Dharmacon, Inc. Fuctional and hyperfunctional sirna
US9771586B2 (en) 2002-11-14 2017-09-26 Thermo Fisher Scientific Inc. RNAi targeting ZNF205
US9719092B2 (en) 2002-11-14 2017-08-01 Thermo Fisher Scientific Inc. RNAi targeting CNTD2
US7619081B2 (en) 2002-11-14 2009-11-17 Dharmacon, Inc. siRNA targeting coatomer protein complex, subunit beta 2 (COPB2)
AU2004235396B2 (en) 2003-04-29 2010-11-25 Sarepta Therapeutics, Inc. Compositions for enhancing transport and antisense efficacy of nucleic acid analog into cells
US20060040882A1 (en) 2004-05-04 2006-02-23 Lishan Chen Compostions and methods for enhancing delivery of nucleic acids into cells and for modifying expression of target genes in cells
US7605250B2 (en) 2004-05-12 2009-10-20 Dharmacon, Inc. siRNA targeting cAMP-specific phosphodiesterase 4D
US8067571B2 (en) 2005-07-13 2011-11-29 Avi Biopharma, Inc. Antibacterial antisense oligonucleotide and method
JP2009507852A (en) * 2005-09-08 2009-02-26 エムディーアールエヌエー，インコーポレイテッド Pharmaceutical composition for delivery of ribonucleic acid to cells
EP1942943A2 (en) * 2005-11-04 2008-07-16 Nastech Pharmaceutical Company Inc. Peptide-dicer substrate rna conjugates as delivery vehicles for sirna
BRPI0620074A2 (en) * 2005-12-19 2011-11-01 Sirna Therapeutics Inc composition
CN101506368B (en) * 2006-07-12 2017-02-08 加利福尼亚大学董事会 Transducible delivery of nucleic acids by reversible phosphotriester charge neutralization protecting groups
LT2056845T (en) 2006-08-08 2017-11-27 Rheinische Friedrich-Wilhelms-Universität Bonn Structure and use of 5` phosphate oligonucleotides
CA2661093A1 (en) * 2006-08-18 2008-02-21 Nastech Pharmaceutical Company Inc. Dicer substrate rna peptide conjugates and methods for rna therapeutics
KR20080076622A (en) * 2007-02-16 2008-08-20 포항공과대학교 산학협력단 Oligomerizaed protein transduction domains and method for the intracellular transduction of viral vectors
US20100016215A1 (en) 2007-06-29 2010-01-21 Avi Biopharma, Inc. Compound and method for treating myotonic dystrophy
TW200932274A (en) 2007-12-18 2009-08-01 Alcon Res Ltd Interfering RNA delivery system and uses thereof
JP2011505846A (en) * 2007-12-20 2011-03-03 アンジオケム，インコーポレーテッド Polypeptide-nucleic acid conjugates and uses thereof
JP5689413B2 (en) 2008-05-21 2015-03-25 ライニッシュ フリードリッヒ−ウィルヘルムズ−ユニバーシタット ボン 5 'triphosphate oligonucleotide having blunt ends and uses thereof
WO2012118911A1 (en) * 2011-03-03 2012-09-07 Quark Pharmaceuticals, Inc. Oligonucleotide modulators of the toll-like receptor pathway
EP2508530A1 (en) 2011-03-28 2012-10-10 Rheinische Friedrich-Wilhelms-Universität Bonn Purification of triphosphorylated oligonucleotides using capture tags
US9161948B2 (en) 2011-05-05 2015-10-20 Sarepta Therapeutics, Inc. Peptide oligonucleotide conjugates
EP2704749A1 (en) * 2011-05-05 2014-03-12 Sarepta Therapeutics, Inc. Peptide oligonucleotide conjugates
AU2013306006B2 (en) 2012-08-20 2017-07-06 The Regents Of The University Of California Polynucleotides having bioreversible groups
EP2712870A1 (en) 2012-09-27 2014-04-02 Rheinische Friedrich-Wilhelms-Universität Bonn Novel RIG-I ligands and methods for producing them
ES2877553T3 (en) * 2013-04-10 2021-11-17 Aarhus Univ University Of Aarhus Use of immunosuppressive domains as drugs
EP3302489A4 (en) 2015-06-04 2019-02-06 Sarepta Therapeutics, Inc. Methods and compounds for treatment of lymphocyte-related diseases and conditions
EP3500581A4 (en) 2016-08-17 2021-10-06 Solstice Biologics, Ltd. Polynucleotide constructs
US11597744B2 (en) 2017-06-30 2023-03-07 Sirius Therapeutics, Inc. Chiral phosphoramidite auxiliaries and methods of their use
CN116949044B (en) * 2023-09-20 2023-12-12 北京炫景瑞医药科技有限公司 Double-stranded oligonucleotide, conjugate comprising double-stranded oligonucleotide and application thereof

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Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US9228186B2 (en) 2002-11-14 2016-01-05 Thermo Fisher Scientific Inc. Methods and compositions for selecting siRNA of improved functionality

Also Published As

Publication number Publication date
CA2565685A1 (en) 2005-12-15
MXPA06012605A (en) 2006-12-15
WO2005117991A2 (en) 2005-12-15
JP2007536253A (en) 2007-12-13
EP1750775A2 (en) 2007-02-14

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