BR0208962A - Método para liberar in vivo um oligonucleotìdeo quimérico para dentro de células alvo de um tecido humano ou animal, para tratar uma doença, para obter um modelo animal, para selecionar compostos cosméticos ou farmacêuticos, para tratar um hospedeiro humano possuindo retinopatia e para tratar um hospedeiro humano ou animal possuindo neovascularização ocular, aligonucleotìdeo quimérico, composição farmacêutica, modelo animal, e, uso do mesmo - Google Patents

Método para liberar in vivo um oligonucleotìdeo quimérico para dentro de células alvo de um tecido humano ou animal, para tratar uma doença, para obter um modelo animal, para selecionar compostos cosméticos ou farmacêuticos, para tratar um hospedeiro humano possuindo retinopatia e para tratar um hospedeiro humano ou animal possuindo neovascularização ocular, aligonucleotìdeo quimérico, composição farmacêutica, modelo animal, e, uso do mesmo

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Publication number
BR0208962A
BR0208962A BR0208962-9A BR0208962A BR0208962A BR 0208962 A BR0208962 A BR 0208962A BR 0208962 A BR0208962 A BR 0208962A BR 0208962 A BR0208962 A BR 0208962A
Authority
BR
Brazil
Prior art keywords
human
animal
treat
chimeric
animal model
Prior art date
Application number
BR0208962-9A
Other languages
English (en)
Inventor
Ther Se Bizemont
Florian Sennlaub
Francine Behar-Cohen
Yves Courtois
Original Assignee
Optis France S A
Inst Nat Sante Rech Med
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 Optis France S A, Inst Nat Sante Rech Med filed Critical Optis France S A
Publication of BR0208962A publication Critical patent/BR0208962A/pt

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Classifications

    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0083—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the administration regime
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/02—Ophthalmic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • 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
    • 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

Abstract

"MéTODOS PARA LIBERAR IN VIVO UM OLIGONUCLEOTìDEO QUIMéRICO PARA DENTRO DE CéLULAS ALVO DE UM TECIDO HUMANO OU ANIMAL, PARA TRATAR UMA DOENçA, PARA OBTER UM MODELO ANIMAL, PARA SELECIONAR COMPOSTOS COSMéTICOS OU FARMACêUTICOS, PARA TRATAR UM HOSPEDEIRO HUMANO POSSUINDO RETINOPATIA E PARA TRATAR UM HOSPEDEIRO HUMANO OU ANIMAL POSSUINDO NEOVASCULARIZAçãO OCULAR, OLIGONUCLEOTìDEO QUIMéRICO, COMPOSIçãO FARMACêUTICA, MODELO ANIMAL, E, USO DO MESMO". A presente invenção proporciona um método para intensificar a liberação in vivo de oligonucleotídeos quiméricos, contendo por exemplo DNA/2<39>OMeRNA, para dentro de células de uma planta, um animal, ou um humano, compreendendo uma etapa de aplicar topicamente em ou de injetar para dentro de um tecido, ou tecido adjacente a um tecido, contendo as citadas células, uma composição compreendendo o citado oligonucleotídeo quimérico, seguida, precedida por, ou simultânea a uma etapa de transferir por iontoforese o citado oligonucleotídeo quimérico para dentro de citadas células, e refere-se a um método de terapia genética compreendendo a transferência por iontoforese de um oligonucleotídeo quimérico DNA/2<39>OMeRNA. A presente invenção também se refere aos oligonucleotídeos quiméricos particulares DNA/2<39>OMeRNA capazes de induzirem ou inibirem a expressão de um gene específico envolvido na função ocular pela indução ou retenção de uma mutação naquele gene específico, e seu uso como composição terapêutica para prevenir ou tratar doenças oculares.
BR0208962-9A 2001-04-17 2002-04-17 Método para liberar in vivo um oligonucleotìdeo quimérico para dentro de células alvo de um tecido humano ou animal, para tratar uma doença, para obter um modelo animal, para selecionar compostos cosméticos ou farmacêuticos, para tratar um hospedeiro humano possuindo retinopatia e para tratar um hospedeiro humano ou animal possuindo neovascularização ocular, aligonucleotìdeo quimérico, composição farmacêutica, modelo animal, e, uso do mesmo BR0208962A (pt)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US09/836,439 US20030045830A1 (en) 2001-04-17 2001-04-17 Gene therapy with chimeric oligonucleotides delivered by a method comprising a step of iontophoresis
PCT/IB2002/002310 WO2002083184A2 (en) 2001-04-17 2002-04-17 Gene therapy with chimeric oligonucleotides delivered by a method comprising a step of iontophoresis

Publications (1)

Publication Number Publication Date
BR0208962A true BR0208962A (pt) 2004-07-27

Family

ID=25271967

Family Applications (1)

Application Number Title Priority Date Filing Date
BR0208962-9A BR0208962A (pt) 2001-04-17 2002-04-17 Método para liberar in vivo um oligonucleotìdeo quimérico para dentro de células alvo de um tecido humano ou animal, para tratar uma doença, para obter um modelo animal, para selecionar compostos cosméticos ou farmacêuticos, para tratar um hospedeiro humano possuindo retinopatia e para tratar um hospedeiro humano ou animal possuindo neovascularização ocular, aligonucleotìdeo quimérico, composição farmacêutica, modelo animal, e, uso do mesmo

Country Status (10)

Country Link
US (2) US20030045830A1 (pt)
EP (1) EP1379665A2 (pt)
JP (1) JP2004532844A (pt)
KR (1) KR20040018356A (pt)
AU (1) AU2002311523A1 (pt)
BR (1) BR0208962A (pt)
CA (1) CA2443923A1 (pt)
IL (1) IL158347A0 (pt)
MX (1) MXPA03009528A (pt)
WO (1) WO2002083184A2 (pt)

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US7517864B2 (en) * 2001-05-18 2009-04-14 Sirna Therapeutics, Inc. RNA interference mediated inhibition of vascular endothelial growth factor and vascular endothelial growth factor receptor gene expression using short interfering nucleic acid (siNA)
EP1495121A2 (en) 2002-04-18 2005-01-12 Lynkeus Biotech GmbH Means and methods for the specific inhibition of genes in cells and tissue of the cns and/or eye
US7148342B2 (en) 2002-07-24 2006-12-12 The Trustees Of The University Of Pennyslvania Compositions and methods for sirna inhibition of angiogenesis
US20070218551A1 (en) * 2003-10-02 2007-09-20 Chuan-Yuan Li Novel Sirna-Based Approach to Target the Hif-Alpha Factor for Gene Therapy
US7840263B2 (en) * 2004-02-27 2010-11-23 Cardiac Pacemakers, Inc. Method and apparatus for device controlled gene expression
US7764995B2 (en) 2004-06-07 2010-07-27 Cardiac Pacemakers, Inc. Method and apparatus to modulate cellular regeneration post myocardial infarct
US7729761B2 (en) * 2004-07-14 2010-06-01 Cardiac Pacemakers, Inc. Method and apparatus for controlled gene or protein delivery
US20070299386A1 (en) * 2006-06-23 2007-12-27 Minu, L.L.C. Delivery of an ocular agent using iontophoresis
US20070299420A1 (en) * 2006-06-23 2007-12-27 Minu, L.L.C. Delivery of an agent using iontophoresis
JP2010511692A (ja) * 2006-12-05 2010-04-15 アイゲート ファーマ エスエーエス イオントフォレシスを通じての核酸の強化された網膜送達
NZ593743A (en) 2008-12-04 2012-07-27 Opko Ophthalmics Llc Compositions and methods for selective inhibition of pro-angiogenic vegf isoforms
US8207138B2 (en) * 2009-05-19 2012-06-26 Medtronic, Inc. Methods and devices for improved efficiency of RNA delivery to cells
EP2559443A1 (en) * 2011-08-16 2013-02-20 INSERM (Institut National de la Santé et de la Recherche Médicale) Methods and pharmaceutical compositions for the treatment of an ocular disease in a subject
CA2845549C (en) * 2011-08-16 2020-07-28 Institut National De La Sante Et De La Recherche Medicale Device for the treatment of an ocular disease
WO2016094845A2 (en) * 2014-12-12 2016-06-16 Woolf Tod M Compositions and methods for editing nucleic acids in cells utilizing oligonucleotides

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Also Published As

Publication number Publication date
US20060031946A1 (en) 2006-02-09
WO2002083184A2 (en) 2002-10-24
KR20040018356A (ko) 2004-03-03
AU2002311523A1 (en) 2002-10-28
JP2004532844A (ja) 2004-10-28
IL158347A0 (en) 2004-05-12
MXPA03009528A (es) 2004-12-06
US20030045830A1 (en) 2003-03-06
WO2002083184A3 (en) 2003-09-18
EP1379665A2 (en) 2004-01-14
CA2443923A1 (en) 2002-10-24

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B06F Objections, documents and/or translations needed after an examination request according [chapter 6.6 patent gazette]
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B08F Application dismissed because of non-payment of annual fees [chapter 8.6 patent gazette]

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