WO2007121326A3 - Compositions and methods for modulating gene expression - Google Patents

Compositions and methods for modulating gene expression Download PDF

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Publication number
WO2007121326A3
WO2007121326A3 PCT/US2007/066571 US2007066571W WO2007121326A3 WO 2007121326 A3 WO2007121326 A3 WO 2007121326A3 US 2007066571 W US2007066571 W US 2007066571W WO 2007121326 A3 WO2007121326 A3 WO 2007121326A3
Authority
WO
WIPO (PCT)
Prior art keywords
atfs
gene expression
compositions
methods
modulating
Prior art date
Application number
PCT/US2007/066571
Other languages
French (fr)
Other versions
WO2007121326A2 (en
Inventor
Dusan Stanojevic
Original Assignee
Crosslink Genetics Corp
Dusan Stanojevic
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 Crosslink Genetics Corp, Dusan Stanojevic filed Critical Crosslink Genetics Corp
Priority to EP07760600A priority Critical patent/EP2015782A4/en
Priority to CA002649114A priority patent/CA2649114A1/en
Publication of WO2007121326A2 publication Critical patent/WO2007121326A2/en
Publication of WO2007121326A3 publication Critical patent/WO2007121326A3/en

Links

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
    • C12N15/111—General methods applicable to biologically active non-coding nucleic acids
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/62—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being a protein, peptide or polyamino acid
    • A61K47/64—Drug-peptide, drug-protein or drug-polyamino acid conjugates, i.e. the modifying agent being a peptide, protein or polyamino acid which is covalently bonded or complexed to a therapeutically active agent
    • 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
    • C12N2310/11—Antisense
    • 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
    • C12N2310/15—Nucleic acids forming more than 2 strands, e.g. TFOs
    • 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
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C12N2310/3513—Protein; Peptide

Abstract

A description of novel artificial transcription factors (ATFs) is provided having a non- peptidic DNA-binding domain, flexible linker, and an effector domain based on a small molecule compound. These ATFs are capable of modulating transcription from nucleic acids both in vitro and in vivo. Importantly, these novel ATFs are capable of targeting and modulating the transcription of native (endogenous) genes in vivo. Method for targeted regulation of gene expression and the development of new class of pharmaceuticals are also provided.
PCT/US2007/066571 2006-04-12 2007-04-12 Compositions and methods for modulating gene expression WO2007121326A2 (en)

Priority Applications (2)

Application Number Priority Date Filing Date Title
EP07760600A EP2015782A4 (en) 2006-04-12 2007-04-12 Compositions and methods for modulating gene expression
CA002649114A CA2649114A1 (en) 2006-04-12 2007-04-12 Compositions and methods for modulating gene expression

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US79130506P 2006-04-12 2006-04-12
US60/791,305 2006-04-12

Publications (2)

Publication Number Publication Date
WO2007121326A2 WO2007121326A2 (en) 2007-10-25
WO2007121326A3 true WO2007121326A3 (en) 2008-12-24

Family

ID=38610393

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US2007/066571 WO2007121326A2 (en) 2006-04-12 2007-04-12 Compositions and methods for modulating gene expression

Country Status (3)

Country Link
EP (1) EP2015782A4 (en)
CA (1) CA2649114A1 (en)
WO (1) WO2007121326A2 (en)

Families Citing this family (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3485912A1 (en) * 2009-04-08 2019-05-22 Faulstich, Heinz, Dr. Amatoxin-armed therapeutic cell surface binding components designed for tumour therapy
ES2402254T3 (en) 2010-09-30 2013-04-30 Heidelberg Pharma Ag Amatoxin conjugates with improved linkers
KR102092981B1 (en) 2012-06-07 2020-03-24 더 칠드런스 호스피탈 오브 필라델피아 Controlled gene expression methods
EP2774624A1 (en) 2013-03-04 2014-09-10 Heidelberg Pharma GmbH Amatoxin derivatives
JP6321687B2 (en) * 2014-03-10 2018-05-09 ハイデルベルク ファルマ ゲゼルシャフト ミット ベシュレンクテル ハフツング Amatoxin derivatives
IL263744B2 (en) 2016-06-17 2023-11-01 Magenta Therapeutics Inc Compositions and methods for the depletion of cd117 plus cells
CN109125736B (en) * 2017-08-18 2022-01-04 四川百利药业有限责任公司 Non-natural amatoxin antibody conjugate
WO2024036143A2 (en) * 2022-08-09 2024-02-15 Ptc Therapeutics, Inc. Methods for modulating rna splicing

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20030105045A1 (en) * 2000-10-13 2003-06-05 Crosslink Genetics Corporation Artificial transcriptional factors and methods of use

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20030105045A1 (en) * 2000-10-13 2003-06-05 Crosslink Genetics Corporation Artificial transcriptional factors and methods of use

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
INGLES C.J. ET AL.: "Alpha-amantin Resistance of RNA Polymerase II in Mutant Chinese Hamster Ovary Cell Lines", THE JOURNAL OF BIOLOGICAL CHEMISTRY, vol. 251, no. 9, May 1976 (1976-05-01), pages 2729 - 2734, XP007911801 *
See also references of EP2015782A4 *

Also Published As

Publication number Publication date
EP2015782A2 (en) 2009-01-21
EP2015782A4 (en) 2010-04-07
CA2649114A1 (en) 2007-10-25
WO2007121326A2 (en) 2007-10-25

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