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WO2003012052A3 - Specific inhibition of gene expression by small double stranded rnas - Google Patents

Specific inhibition of gene expression by small double stranded rnas Download PDF

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Publication number
WO2003012052A3
WO2003012052A3 PCT/US2002/024226 US0224226W WO03012052A3 WO 2003012052 A3 WO2003012052 A3 WO 2003012052A3 US 0224226 W US0224226 W US 0224226W WO 03012052 A3 WO03012052 A3 WO 03012052A3
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WO
WIPO (PCT)
Prior art keywords
double stranded
stranded rnas
rnas
expression
small double
Prior art date
Application number
PCT/US2002/024226
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French (fr)
Other versions
WO2003012052A2 (en
Inventor
Natasha J Caplen
Richard A Morgan
Andrew Fire
Susan Parrish
Spyro Mousses
Olli Kallioniemi
J Robert Cornelison
Eric Wfw Alton
Uta Griesenbach
Original Assignee
Us Gov Health & Human Serv
Carnegie Inst Of Washington
Imp College Innovatuions Ltd
Natasha J Caplen
Richard A Morgan
Andrew Fire
Susan Parrish
Spyro Mousses
Olli Kallioniemi
J Robert Cornelison
Eric Wfw Alton
Uta Griesenbach
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 Us Gov Health & Human Serv, Carnegie Inst Of Washington, Imp College Innovatuions Ltd, Natasha J Caplen, Richard A Morgan, Andrew Fire, Susan Parrish, Spyro Mousses, Olli Kallioniemi, J Robert Cornelison, Eric Wfw Alton, Uta Griesenbach filed Critical Us Gov Health & Human Serv
Priority to AU2002329667A priority Critical patent/AU2002329667A1/en
Publication of WO2003012052A2 publication Critical patent/WO2003012052A2/en
Publication of WO2003012052A3 publication Critical patent/WO2003012052A3/en

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    • 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
    • C12N15/1138—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 receptors or cell surface proteins
    • C—CHEMISTRY; METALLURGY
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    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • C12N2310/00—Structure or type of the nucleic acid
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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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    • 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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    • C12N2320/00—Applications; Uses
    • C12N2320/50—Methods for regulating/modulating their activity
    • C12N2320/51—Methods for regulating/modulating their activity modulating the chemical stability, e.g. nuclease-resistance

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  • Wood Science & Technology (AREA)
  • Microbiology (AREA)
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  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Physics & Mathematics (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)

Abstract

Synthetic small double stranded RNAs that can induce gene specific inhibition of expression are provided, as are methods for selecting and constructing optimal small double-stranded RNAs. Provided RNAs are suitable for interference or inhibition of expression of a target gene and comprise double stranded RNAs of about to about 40 nucleotides containing a 3' and/or 5' overhang on each strand having a length of O-nucleotide to 5-nucleotides, wherein the sequence of the double stranded RNAs are substantially identical (e.g., differing by no more than 30%, or more preferably no more than 10% ) to a portion of a mRNA or transcript of the target gene for which interference or inhibition of expression is desired. The current disclosure is also directed to the use of such synthetic small double stranded RNAs as reverse genetic and/or therapeutic tools. Also provided are methods, including high-throughput methods, of testing, analyzing, and optimizing short double-stranded 15 RNAs for use in gene specific inhibition of expression.
PCT/US2002/024226 2001-07-30 2002-07-30 Specific inhibition of gene expression by small double stranded rnas WO2003012052A2 (en)

Priority Applications (1)

Application Number Priority Date Filing Date Title
AU2002329667A AU2002329667A1 (en) 2001-07-30 2002-07-30 Specific inhibition of gene expression by small double stranded rnas

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US30864001P 2001-07-30 2001-07-30
US60/308,640 2001-07-30
US37097002P 2002-04-08 2002-04-08
US60/370,970 2002-04-08

Publications (2)

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WO2003012052A2 WO2003012052A2 (en) 2003-02-13
WO2003012052A3 true WO2003012052A3 (en) 2005-03-31

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Country Status (2)

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AU (1) AU2002329667A1 (en)
WO (1) WO2003012052A2 (en)

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US9109229B2 (en) 2004-07-26 2015-08-18 Pfenex Inc. Process for improved protein expression by strain engineering
US9394571B2 (en) 2007-04-27 2016-07-19 Pfenex Inc. Method for rapidly screening microbial hosts to identify certain strains with improved yield and/or quality in the expression of heterologous proteins
US9453251B2 (en) 2002-10-08 2016-09-27 Pfenex Inc. Expression of mammalian proteins in Pseudomonas fluorescens
US9580719B2 (en) 2007-04-27 2017-02-28 Pfenex, Inc. Method for rapidly screening microbial hosts to identify certain strains with improved yield and/or quality in the expression of heterologous proteins

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Cited By (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US9453251B2 (en) 2002-10-08 2016-09-27 Pfenex Inc. Expression of mammalian proteins in Pseudomonas fluorescens
US9458487B2 (en) 2002-10-08 2016-10-04 Pfenex, Inc. Expression of mammalian proteins in pseudomonas fluorescens
US9109229B2 (en) 2004-07-26 2015-08-18 Pfenex Inc. Process for improved protein expression by strain engineering
US9394571B2 (en) 2007-04-27 2016-07-19 Pfenex Inc. Method for rapidly screening microbial hosts to identify certain strains with improved yield and/or quality in the expression of heterologous proteins
US9580719B2 (en) 2007-04-27 2017-02-28 Pfenex, Inc. Method for rapidly screening microbial hosts to identify certain strains with improved yield and/or quality in the expression of heterologous proteins

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AU2002329667A1 (en) 2003-02-17

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