ITTO20090487A1 - ANTISENSE OLIGONUCLEOTIDS ACTS TO INDOCATE EXONIC SKIPPING AND THEIR USE AS MEDICATION FOR THE TREATMENT OF DUCHENNE MUSCULAR DYSTROPHY (DMD) - Google Patents

ANTISENSE OLIGONUCLEOTIDS ACTS TO INDOCATE EXONIC SKIPPING AND THEIR USE AS MEDICATION FOR THE TREATMENT OF DUCHENNE MUSCULAR DYSTROPHY (DMD)

Info

Publication number
ITTO20090487A1
ITTO20090487A1 IT000487A ITTO20090487A ITTO20090487A1 IT TO20090487 A1 ITTO20090487 A1 IT TO20090487A1 IT 000487 A IT000487 A IT 000487A IT TO20090487 A ITTO20090487 A IT TO20090487A IT TO20090487 A1 ITTO20090487 A1 IT TO20090487A1
Authority
IT
Italy
Prior art keywords
indocate
exonic
dmd
skipping
medication
Prior art date
Application number
IT000487A
Other languages
Italian (it)
Inventor
Alessandra Ferlini
Alessandro Medici
Daniela Perrone
Paola Rimessi
Pietro Spitali
Original Assignee
Univ Ferrara
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 Univ Ferrara filed Critical Univ Ferrara
Priority to IT000487A priority Critical patent/ITTO20090487A1/en
Priority to PCT/IB2010/052912 priority patent/WO2010150231A1/en
Publication of ITTO20090487A1 publication Critical patent/ITTO20090487A1/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
    • C12N15/113—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
    • 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
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
  • Molecular Biology (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Biophysics (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
IT000487A 2009-06-26 2009-06-26 ANTISENSE OLIGONUCLEOTIDS ACTS TO INDOCATE EXONIC SKIPPING AND THEIR USE AS MEDICATION FOR THE TREATMENT OF DUCHENNE MUSCULAR DYSTROPHY (DMD) ITTO20090487A1 (en)

Priority Applications (2)

Application Number Priority Date Filing Date Title
IT000487A ITTO20090487A1 (en) 2009-06-26 2009-06-26 ANTISENSE OLIGONUCLEOTIDS ACTS TO INDOCATE EXONIC SKIPPING AND THEIR USE AS MEDICATION FOR THE TREATMENT OF DUCHENNE MUSCULAR DYSTROPHY (DMD)
PCT/IB2010/052912 WO2010150231A1 (en) 2009-06-26 2010-06-25 Antisense oligonucleotides capable of inducing exon skipping and the use thereof as a medicament for the treatment of duchenne muscular dystrophy (dmd)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
IT000487A ITTO20090487A1 (en) 2009-06-26 2009-06-26 ANTISENSE OLIGONUCLEOTIDS ACTS TO INDOCATE EXONIC SKIPPING AND THEIR USE AS MEDICATION FOR THE TREATMENT OF DUCHENNE MUSCULAR DYSTROPHY (DMD)

Publications (1)

Publication Number Publication Date
ITTO20090487A1 true ITTO20090487A1 (en) 2010-12-27

Family

ID=41508769

Family Applications (1)

Application Number Title Priority Date Filing Date
IT000487A ITTO20090487A1 (en) 2009-06-26 2009-06-26 ANTISENSE OLIGONUCLEOTIDS ACTS TO INDOCATE EXONIC SKIPPING AND THEIR USE AS MEDICATION FOR THE TREATMENT OF DUCHENNE MUSCULAR DYSTROPHY (DMD)

Country Status (2)

Country Link
IT (1) ITTO20090487A1 (en)
WO (1) WO2010150231A1 (en)

Families Citing this family (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
USRE48960E1 (en) 2004-06-28 2022-03-08 The University Of Western Australia Antisense oligonucleotides for inducing exon skipping and methods of use thereof
ATE498685T1 (en) 2004-06-28 2011-03-15 Univ Western Australia ANTISENSE OLIGONUCLEOTIDES FOR INDUCING EXON SKIPPING AND METHOD FOR THE USE THEREOF
CN105779453A (en) 2008-10-24 2016-07-20 萨雷普塔治疗公司 Multiple exon skipping compositions for DMD
KR102113306B1 (en) 2009-11-12 2020-05-21 더 유니버시티 오브 웨스턴 오스트레일리아 Antisense Molecules and Methods for Treating Pathologies
KR102390965B1 (en) 2013-03-14 2022-04-26 사렙타 쎄러퓨틱스 인코퍼레이티드 Exon skipping compositions for treating muscular dystrophy
CN113633787A (en) 2013-03-15 2021-11-12 萨勒普塔医疗公司 Improved composition for treating muscular dystrophy
KR20220053048A (en) * 2015-09-15 2022-04-28 니뽄 신야쿠 가부시키가이샤 Antisense nucleic acids

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2006000057A1 (en) * 2004-06-28 2006-01-05 SMITHKLINE BEECHAM CORPORATION, doing business as GLAXOSMITHKLINE Antisense oligonucleotides for inducing exon skipping and methods of use thereof
WO2007135105A1 (en) * 2006-05-19 2007-11-29 Academisch Ziekenhuis Leiden Means and method for inducing exon-skipping
WO2009101399A1 (en) * 2008-02-12 2009-08-20 Isis Innovation Limited Treatment of muscular dystrophy using peptide nucleic acid ( pna)

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2006000057A1 (en) * 2004-06-28 2006-01-05 SMITHKLINE BEECHAM CORPORATION, doing business as GLAXOSMITHKLINE Antisense oligonucleotides for inducing exon skipping and methods of use thereof
WO2007135105A1 (en) * 2006-05-19 2007-11-29 Academisch Ziekenhuis Leiden Means and method for inducing exon-skipping
WO2009101399A1 (en) * 2008-02-12 2009-08-20 Isis Innovation Limited Treatment of muscular dystrophy using peptide nucleic acid ( pna)

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
AARTSMA-RUS A. ET AL.: "Entries in the Leiden Duchenne Muscular Distrophy mutation database: an overview of mutation types and paradoxical cases that confirm the reading-frame rule", MUSCLE & NERVE, vol. 34, 2006, pages 135 - 144, XP002564020 *
AARTSMA-RUS A. ET AL: "Theoretic applicability of antisense-mediated exon skipping for Duchenne Muscular Dystrophy mutations", HUMAN MUTATION, JOHN WILEY & SONS, INC, US, vol. 30, no. 3, 1 March 2009 (2009-03-01), pages 293 - 299, XP002541505, ISSN: 1059-7794, [retrieved on 20090120] *

Also Published As

Publication number Publication date
WO2010150231A1 (en) 2010-12-29

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