WO2005045037A3 - RNA INTERFERENCE MEDIATED INHIBITION OF 5-ALPHA REDUCTASE AND ANDROGEN RECEPTOR GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA) - Google Patents

RNA INTERFERENCE MEDIATED INHIBITION OF 5-ALPHA REDUCTASE AND ANDROGEN RECEPTOR GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA) Download PDF

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Publication number
WO2005045037A3
WO2005045037A3 PCT/US2004/027169 US2004027169W WO2005045037A3 WO 2005045037 A3 WO2005045037 A3 WO 2005045037A3 US 2004027169 W US2004027169 W US 2004027169W WO 2005045037 A3 WO2005045037 A3 WO 2005045037A3
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WIPO (PCT)
Prior art keywords
nucleic acid
androgen receptor
short interfering
sina
alpha reductase
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PCT/US2004/027169
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French (fr)
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WO2005045037A2 (en
WO2005045037B1 (en
Inventor
James Mcswiggen
Narendra Vaish
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Sirna Therapeutics Inc
James Mcswiggen
Narendra Vaish
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 US10/693,059 external-priority patent/US20080039414A1/en
Priority claimed from US10/720,448 external-priority patent/US8273866B2/en
Priority claimed from US10/727,780 external-priority patent/US20050233329A1/en
Priority claimed from US10/757,803 external-priority patent/US20050020525A1/en
Priority claimed from US10/780,447 external-priority patent/US7491805B2/en
Priority claimed from US10/826,966 external-priority patent/US20050032733A1/en
Priority claimed from PCT/US2004/013456 external-priority patent/WO2005041859A2/en
Priority claimed from US10/844,072 external-priority patent/US20050159376A1/en
Priority claimed from PCT/US2004/016390 external-priority patent/WO2005019453A2/en
Application filed by Sirna Therapeutics Inc, James Mcswiggen, Narendra Vaish filed Critical Sirna Therapeutics Inc
Publication of WO2005045037A2 publication Critical patent/WO2005045037A2/en
Publication of WO2005045037A3 publication Critical patent/WO2005045037A3/en
Publication of WO2005045037B1 publication Critical patent/WO2005045037B1/en

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    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
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    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • C12N15/1137Non-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 enzymes
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    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • C12N15/1138Non-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
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    • C12Y103/00Oxidoreductases acting on the CH-CH group of donors (1.3)
    • C12Y103/01Oxidoreductases acting on the CH-CH group of donors (1.3) with NAD+ or NADP+ as acceptor (1.3.1)
    • C12Y103/010223-Oxo-5alpha-steroid 4-dehydrogenase (NADP+) (1.3.1.22), i.e. cortisone alpha-reductase
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • C12N2310/111Antisense spanning the whole gene, or a large part of it
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    • C12N2310/34Spatial arrangement of the modifications
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    • C12N2310/50Physical structure
    • C12N2310/53Physical structure partially self-complementary or closed

Abstract

This invention relates to compounds, compositions, and methods useful for modulating 5-alpha reductase (SRD5A-1, SRD5A-2) and androgen receptor (AR) 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 5-alpha reductase and androgen receptor (AR) 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 5-alpha reductase and androgen receptor (AR) genes.
PCT/US2004/027169 2003-10-23 2004-08-20 RNA INTERFERENCE MEDIATED INHIBITION OF 5-ALPHA REDUCTASE AND ANDROGEN RECEPTOR GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA) WO2005045037A2 (en)

Applications Claiming Priority (20)

Application Number Priority Date Filing Date Title
US10/693,059 2003-10-23
US10/693,059 US20080039414A1 (en) 2002-02-20 2003-10-23 RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (siNA)
US10/720,448 2003-11-24
US10/720,448 US8273866B2 (en) 2002-02-20 2003-11-24 RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (SINA)
US10/727,780 US20050233329A1 (en) 2002-02-20 2003-12-03 Inhibition of gene expression using duplex forming oligonucleotides
US10/727,780 2003-12-03
US10/757,803 2004-01-14
US10/757,803 US20050020525A1 (en) 2002-02-20 2004-01-14 RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (siNA)
US54348004P 2004-02-10 2004-02-10
US60/543,480 2004-02-10
US10/780,447 US7491805B2 (en) 2001-05-18 2004-02-13 Conjugates and compositions for cellular delivery
US10/780.447 2004-02-13
US10/826,966 2004-04-16
US10/826,966 US20050032733A1 (en) 2001-05-18 2004-04-16 RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (SiNA)
USPCT/US04/13456 2004-04-30
PCT/US2004/013456 WO2005041859A2 (en) 2003-04-30 2004-04-30 Conjugates and compositions for cellular delivery.
US10/844,072 US20050159376A1 (en) 2002-02-20 2004-05-12 RNA interference mediated inhibition 5-alpha reductase and androgen receptor gene expression using short interfering nucleic acid (siNA)
US10/844,072 2004-05-12
USPCT/US04/16390 2004-05-24
PCT/US2004/016390 WO2005019453A2 (en) 2001-05-18 2004-05-24 RNA INTERFERENCE MEDIATED INHIBITION OF GENE EXPRESSION USING CHEMICALLY MODIFIED SHORT INTERFERING NUCLEIC ACID (siNA)

Publications (3)

Publication Number Publication Date
WO2005045037A2 WO2005045037A2 (en) 2005-05-19
WO2005045037A3 true WO2005045037A3 (en) 2005-11-03
WO2005045037B1 WO2005045037B1 (en) 2005-12-29

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PCT/US2004/027169 WO2005045037A2 (en) 2003-10-23 2004-08-20 RNA INTERFERENCE MEDIATED INHIBITION OF 5-ALPHA REDUCTASE AND ANDROGEN RECEPTOR GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (siNA)

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Cited By (1)

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CN110536965B (en) * 2017-02-21 2023-08-18 奥利克斯医药有限公司 Asymmetric siRNA for inhibiting expression of male pattern alopecia target gene

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PL1781787T3 (en) 2004-08-23 2018-01-31 Sylentis Sau Treatment of eye disorders characterized by an elevated intraocular pressure by sirnas
WO2006024880A2 (en) 2004-08-31 2006-03-09 Sylentis S.A.U. Methods and compositions to inhibit p2x7 receptor expression
GB0505081D0 (en) 2005-03-14 2005-04-20 Genomica Sau Downregulation of interleukin-12 expression by means of rnai technology
GB0521351D0 (en) 2005-10-20 2005-11-30 Genomica Sau Modulation of TRPV expression levels
GB0521716D0 (en) 2005-10-25 2005-11-30 Genomica Sau Modulation of 11beta-hydroxysteriod dehydrogenase 1 expression for the treatment of ocular diseases
GB0605337D0 (en) 2006-03-17 2006-04-26 Genomica Sau Treatment of CNS conditions
US20100292090A1 (en) 2006-08-25 2010-11-18 Oncotherapy Science, Inc. Prognostic markers and therapeutic targets for lung cancer
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WO2008109355A1 (en) * 2007-03-02 2008-09-12 Mdrna, Inc. Nucleic acid compounds for inhibiting srd5a2 gene expression and uses thereof
KR20100075858A (en) 2007-08-24 2010-07-05 온코세라피 사이언스 가부시키가이샤 Cancer-related genes, cdca5, epha7, stk31 and wdhd1
KR20100075452A (en) 2007-08-24 2010-07-02 온코세라피 사이언스 가부시키가이샤 Pkib and naaladl2 for target genes of prostate cancer therapy and diagnosis
WO2010023877A1 (en) 2008-08-27 2010-03-04 Oncotherapy Science, Inc. Prmt1 for target genes of cancer therapy and diagnosis
WO2013036879A1 (en) * 2011-09-08 2013-03-14 Gradalis, Inc. Compositions and methods for treating prostate cancer
GB201215857D0 (en) 2012-09-05 2012-10-24 Sylentis Sau siRNA and their use in methods and compositions for the treatment and/or prevention of eye conditions
CN104781402A (en) 2012-09-05 2015-07-15 西伦蒂斯私人股份公司 siRNA and their use in methods and compositions for the treatment and/or prevention of eye conditions
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WO2015132303A1 (en) 2014-03-04 2015-09-11 Sylentis Sau Sirnas and their use in methods and compositions for the treatment and/or prevention of eye conditions
MX2018002158A (en) 2015-08-25 2018-07-06 Alnylam Pharmaceuticals Inc Methods and compositions for treating a proprotein convertase subtilisin kexin (pcsk9) gene-associated disorder.
KR102473989B1 (en) 2018-11-28 2022-12-07 (주)바이오니아 Double-stranded oligo nucleotide structure comprising androgen receptor specific sequences and composition comprising the same for preventing hair loss and hair growth

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WO1996002256A1 (en) * 1994-07-15 1996-02-01 City Of Hope CLEAVAGE OF 5α-REDUCTASE MRNA
WO1997038728A1 (en) * 1996-04-15 1997-10-23 Dyad Pharmaceutical Corporation Combination therapy for androgenic alopecia with antisense oligonucleotides and minoxidil
WO2000044895A1 (en) * 1999-01-30 2000-08-03 Roland Kreutzer Method and medicament for inhibiting the expression of a defined gene
WO2003070918A2 (en) * 2002-02-20 2003-08-28 Ribozyme Pharmaceuticals, Incorporated Rna interference by modified short interfering nucleic acid
WO2004063331A2 (en) * 2003-01-03 2004-07-29 Gencia Corporation SiRNA MEDIATED POST-TRANSRIPTIONAL GENE SILENCING OF GENES INVOLVED IN ALOPECIA

Patent Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO1996002256A1 (en) * 1994-07-15 1996-02-01 City Of Hope CLEAVAGE OF 5α-REDUCTASE MRNA
WO1997038728A1 (en) * 1996-04-15 1997-10-23 Dyad Pharmaceutical Corporation Combination therapy for androgenic alopecia with antisense oligonucleotides and minoxidil
WO2000044895A1 (en) * 1999-01-30 2000-08-03 Roland Kreutzer Method and medicament for inhibiting the expression of a defined gene
WO2003070918A2 (en) * 2002-02-20 2003-08-28 Ribozyme Pharmaceuticals, Incorporated Rna interference by modified short interfering nucleic acid
WO2004063331A2 (en) * 2003-01-03 2004-07-29 Gencia Corporation SiRNA MEDIATED POST-TRANSRIPTIONAL GENE SILENCING OF GENES INVOLVED IN ALOPECIA

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN110536965B (en) * 2017-02-21 2023-08-18 奥利克斯医药有限公司 Asymmetric siRNA for inhibiting expression of male pattern alopecia target gene

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