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AU2002232536A1 - Chimeric molecules to modulate gene expression - Google Patents

Chimeric molecules to modulate gene expression

Info

Publication number
AU2002232536A1
AU2002232536A1 AU2002232536A AU3253602A AU2002232536A1 AU 2002232536 A1 AU2002232536 A1 AU 2002232536A1 AU 2002232536 A AU2002232536 A AU 2002232536A AU 3253602 A AU3253602 A AU 3253602A AU 2002232536 A1 AU2002232536 A1 AU 2002232536A1
Authority
AU
Australia
Prior art keywords
gene expression
chimeric molecules
modulate gene
nucleic acid
base
Prior art date
Legal status (The legal status 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 status listed.)
Abandoned
Application number
AU2002232536A
Inventor
Luca Cartegni
Adrian R. Krainer
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Cold Spring Harbor Laboratory
Original Assignee
Cold Spring Harbor Laboratory
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 Cold Spring Harbor Laboratory filed Critical Cold Spring Harbor Laboratory
Publication of AU2002232536A1 publication Critical patent/AU2002232536A1/en
Abandoned legal-status Critical Current

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
    • C12N15/113—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
    • 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
    • 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
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • 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
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/318—Chemical structure of the backbone where the PO2 is completely replaced, e.g. MMI or formacetal
    • C12N2310/3181—Peptide nucleic acid, PNA
    • 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
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/323—Chemical structure of the sugar modified ring structure
    • C12N2310/3233—Morpholino-type ring
    • 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
    • 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
    • C12N2310/52—Physical structure branched

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Physics & Mathematics (AREA)
  • Oncology (AREA)
  • Peptides Or Proteins (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)

Abstract

The present invention provides a chimeric molecule including a base-pairing segment that binds specifically to a single-stranded nucleic acid molecule; and a moiety that modulates splicing or translation. The invention also provides a chimeric molecule including a base-pairing segment that binds specifically to a double-stranded nucleic acid molecule; and a peptide that modulates transcription, wherein the peptide comprises up to about one hundred amino acid residues.
AU2002232536A 2000-11-09 2001-11-09 Chimeric molecules to modulate gene expression Abandoned AU2002232536A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US30418200P 2000-11-09 2000-11-09
US60/304,182 2000-11-09
PCT/US2001/047523 WO2002038738A2 (en) 2000-11-09 2001-11-09 Chimeric molecules to modulate gene expression

Publications (1)

Publication Number Publication Date
AU2002232536A1 true AU2002232536A1 (en) 2002-05-21

Family

ID=23175431

Family Applications (1)

Application Number Title Priority Date Filing Date
AU2002232536A Abandoned AU2002232536A1 (en) 2000-11-09 2001-11-09 Chimeric molecules to modulate gene expression

Country Status (6)

Country Link
US (3) US20050054836A1 (en)
EP (1) EP1416972B1 (en)
AT (1) ATE544473T1 (en)
AU (1) AU2002232536A1 (en)
CA (1) CA2437942C (en)
WO (1) WO2002038738A2 (en)

Families Citing this family (48)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US8005621B2 (en) * 2004-09-13 2011-08-23 Agency For Science Technology And Research Transcript mapping method
US7879992B2 (en) 2005-01-31 2011-02-01 Isis Pharmaceuticals, Inc. Modification of MyD88 splicing using modified oligonucleotides
SI3308788T1 (en) 2005-06-23 2019-01-31 Biogen Ma Inc. Compositions and methods for modulation of smn2 splicing
EP1937312B1 (en) * 2005-08-30 2016-06-29 Ionis Pharmaceuticals, Inc. Chimeric oligomeric compounds for modulation of splicing
CA2658683A1 (en) * 2006-07-21 2008-01-24 Centre National De La Recherche Scientifique (Cnrs) Positive cytomodulines to improve bioreactor productivity
EP2219680A2 (en) * 2007-11-13 2010-08-25 Isis Pharmaceuticals, Inc. Compounds and methods for modulating protein expression
WO2009151546A2 (en) * 2008-05-27 2009-12-17 Ptc Therapeutics, Inc. Methods for treating spinal muscular atrophy
US8986935B2 (en) * 2008-08-13 2015-03-24 Ptc Therapeutics, Inc. Methods for treating spinal muscular atrophy
WO2010075303A1 (en) * 2008-12-23 2010-07-01 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Splicing factors with a puf protein rna-binding domain and a splicing effector domain and uses of same
WO2010091308A2 (en) 2009-02-06 2010-08-12 Isis Pharmaceuticals, Inc. Oligomeric compounds and methods
CN115227710A (en) 2009-06-17 2022-10-25 冷泉港实验室 Compositions and methods for modulating SMN2 splicing in a subject
WO2011103215A1 (en) * 2010-02-16 2011-08-25 Mount Sinai School Of Medicine Embedded chimeric peptide nucleic acids and use thereof
US8927502B2 (en) 2010-02-16 2015-01-06 Icahn School Of Medicine At Mount Sinai Embedded chimeric peptide nucleic acids and uses thereof
US9328346B2 (en) 2010-11-12 2016-05-03 The General Hospital Corporation Polycomb-associated non-coding RNAs
US9920317B2 (en) 2010-11-12 2018-03-20 The General Hospital Corporation Polycomb-associated non-coding RNAs
JP5959066B2 (en) 2011-02-17 2016-08-02 セラヴァンス バイオファーマ アール＆ディー アイピー， エルエルシー Substituted aminobutyric acid derivatives as neprilysin inhibitors
CA2848753C (en) 2011-09-14 2022-07-26 Rana Therapeutics, Inc. Multimeric oligonucleotide compounds
US10059941B2 (en) 2012-05-16 2018-08-28 Translate Bio Ma, Inc. Compositions and methods for modulating SMN gene family expression
AU2013262709A1 (en) 2012-05-16 2015-01-22 Rana Therapeutics, Inc. Compositions and methods for modulating MECP2 expression
EP2850188A4 (en) 2012-05-16 2016-01-20 Rana Therapeutics Inc COMPOSITIONS AND METHODS FOR MODULATING THE EXPRESSION OF THE MULTIGENIC FAMILY OF HEMOGLOBIN
US10837014B2 (en) 2012-05-16 2020-11-17 Translate Bio Ma, Inc. Compositions and methods for modulating SMN gene family expression
EA201492122A1 (en) 2012-05-16 2015-10-30 Рана Терапьютикс, Инк. COMPOSITIONS AND METHODS FOR MODULATING UTRN EXPRESSION
US10174323B2 (en) 2012-05-16 2019-01-08 The General Hospital Corporation Compositions and methods for modulating ATP2A2 expression
AU2013315225B2 (en) 2012-09-14 2018-11-08 Translate Bio Ma, Inc. Multimeric oligonucleotide compounds
EP2943225A4 (en) 2013-01-09 2016-07-13 Ionis Pharmaceuticals Inc COMPOSITIONS AND METHODS FOR MODULATION OF SMN2 SPLICING IN A SUBJECT
WO2014172698A1 (en) 2013-04-19 2014-10-23 Isis Pharmaceuticals, Inc. Compositions and methods for modulation nucleic acids through nonsense mediated decay
WO2015023941A1 (en) 2013-08-16 2015-02-19 Rana Therapeutics, Inc. Oligonucleotides targeting euchromatin regions of genes
US10174328B2 (en) 2013-10-04 2019-01-08 Translate Bio Ma, Inc. Compositions and methods for treating amyotrophic lateral sclerosis
MA39835A (en) 2014-04-17 2017-02-22 Biogen Ma Inc Compositions and methods for modulation of smn2 splicing in a subject
BR112017004056A2 (en) 2014-09-12 2017-12-05 Biogen Ma Inc compositions and methods for detecting smn protein in an individual and treating an individual
CA2966044A1 (en) 2014-10-30 2016-05-06 The General Hospital Corporation Methods for modulating atrx-dependent gene repression
US10410118B2 (en) 2015-03-13 2019-09-10 Deep Genomics Incorporated System and method for training neural networks
US10900036B2 (en) 2015-03-17 2021-01-26 The General Hospital Corporation RNA interactome of polycomb repressive complex 1 (PRC1)
US10185803B2 (en) 2015-06-15 2019-01-22 Deep Genomics Incorporated Systems and methods for classifying, prioritizing and interpreting genetic variants and therapies using a deep neural network
EA201800367A1 (en) 2015-12-10 2019-02-28 ПиТиСи ТЕРАПЬЮТИКС, ИНК. METHODS OF TREATMENT OF HUNTINGTON'S DISEASE
US11198867B2 (en) 2016-06-16 2021-12-14 Ionis Pharmaceuticals, Inc. Combinations for the modulation of SMN expression
TW201839136A (en) 2017-02-06 2018-11-01 瑞士商諾華公司 Composition and method for treating hemochromatosis
JP7376471B2 (en) 2017-06-05 2023-11-08 ピーティーシー セラピューティクス， インコーポレイテッド Compounds to treat Huntington's disease
BR112019027717A2 (en) 2017-06-28 2020-07-28 Ptc Therapeutics, Inc. methods to treat huntington's disease
US11395822B2 (en) 2017-06-28 2022-07-26 Ptc Therapeutics, Inc. Methods for treating Huntington's disease
AU2019243048A1 (en) 2018-03-27 2020-10-15 Ptc Therapeutics, Inc. Compounds for treating Huntington's disease
BR112020026534A2 (en) 2018-06-27 2021-03-23 Ptc Therapeutics, Inc. HETEROARYL COMPOUNDS FOR THE TREATMENT OF HUNTINGTON'S DISEASE
SI3814357T1 (en) 2018-06-27 2024-09-30 Ptc Therapeutics, Inc. Heterocyclic and heteroaryl compounds for treating huntington's disease
SG11202111597UA (en) * 2019-04-24 2021-11-29 Stoke Therapeutics Inc Methods and compositions for modulating splicing and translation
BR112021022183A2 (en) 2019-05-05 2021-12-21 Splisense Ltd Synthetic oligonucleotide molecule, pharmaceutical composition, method for the treatment of cystic fibrosis and kit
WO2021084495A1 (en) 2019-11-01 2021-05-06 Novartis Ag The use of a splicing modulator for a treatment slowing progression of huntington's disease
IL295605A (en) 2020-02-28 2022-10-01 Ionis Pharmaceuticals Inc Compounds and methods for modulating smn2
TW202304446A (en) 2021-03-29 2023-02-01 瑞士商諾華公司 The use of a splicing modulator for a treatment slowing progression of huntington’s disease

Family Cites Families (18)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5874555A (en) * 1987-10-30 1999-02-23 California Institute Of Technology Triple helices and processes for making same
US6153737A (en) * 1990-01-11 2000-11-28 Isis Pharmaceuticals, Inc. Derivatized oligonucleotides having improved uptake and other properties
DK51092D0 (en) * 1991-05-24 1992-04-15 Ole Buchardt OLIGONUCLEOTIDE ANALOGUE DESCRIBED BY PEN, MONOMERIC SYNTHONES AND PROCEDURES FOR PREPARING THEREOF, AND APPLICATIONS THEREOF
US5641625A (en) * 1992-05-22 1997-06-24 Isis Pharmaceuticals, Inc. Cleaving double-stranded DNA with peptide nucleic acids
US5539082A (en) * 1993-04-26 1996-07-23 Nielsen; Peter E. Peptide nucleic acids
US6350853B1 (en) * 1993-04-26 2002-02-26 Peter E. Nielsen Conjugated peptide nucleic acids having enhanced cellular uptake
JP3053539B2 (en) * 1995-01-19 2000-06-19 科学技術振興事業団 CDNA for DOCK180 protein gene
US6090947A (en) * 1996-02-26 2000-07-18 California Institute Of Technology Method for the synthesis of pyrrole and imidazole carboxamides on a solid support
US5998140A (en) * 1996-07-31 1999-12-07 The Scripps Research Institute Complex formation between dsDNA and oligomer of cyclic heterocycles
US6265388B1 (en) * 1997-03-21 2001-07-24 President And Fellows Of Harvard College Antisense inhibition of angiogenin expression
US6265382B1 (en) * 1997-04-11 2001-07-24 Warner-Lambert Company Dipeptide inhibitors of protein farnesyltransferase
US6746868B1 (en) * 1997-09-18 2004-06-08 Isis Pharmaceuticals, Inc. Chemical modification of DNA using peptide nucleic acid conjugates
US6395472B1 (en) * 1999-02-05 2002-05-28 Abbott Laboratories Methods of utilizing the TT virus
US6656730B1 (en) * 1999-06-15 2003-12-02 Isis Pharmaceuticals, Inc. Oligonucleotides conjugated to protein-binding drugs
US6033910A (en) * 1999-07-19 2000-03-07 Isis Pharmaceuticals Inc. Antisense inhibition of MAP kinase kinase 6 expression
US6559279B1 (en) * 2000-09-08 2003-05-06 Isis Pharmaceuticals, Inc. Process for preparing peptide derivatized oligomeric compounds
EP1386004A4 (en) * 2001-04-05 2005-02-16 Ribozyme Pharm Inc Modulation of gene expression associated with inflammation proliferation and neurite outgrowth, using nucleic acid based technologies
BRPI0313202A8 (en) * 2002-08-05 2016-08-16 Atugen Ag ADDITIONAL WAYS TO INTERFERE WITH RNA MOLECULES

Also Published As

Publication number Publication date
EP1416972A4 (en) 2004-09-22
ATE544473T1 (en) 2012-02-15
CA2437942C (en) 2013-06-11
EP1416972B1 (en) 2012-02-08
WO2002038738A2 (en) 2002-05-16
CA2437942A1 (en) 2002-05-16
US20110159587A1 (en) 2011-06-30
WO2002038738A3 (en) 2004-02-19
US20050054836A1 (en) 2005-03-10
US20140011977A1 (en) 2014-01-09
EP1416972A2 (en) 2004-05-12

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