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
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
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)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
US7807816B2 (en) | 2004-06-28 | 2010-10-05 | University Of Western Australia | Antisense oligonucleotides for inducing exon skipping and methods of use thereof |
USRE48960E1 (en) | 2004-06-28 | 2022-03-08 | The University Of Western Australia | Antisense oligonucleotides for inducing exon skipping and methods of use thereof |
EP2350281B1 (en) | 2008-10-24 | 2014-05-14 | Sarepta Therapeutics, Inc. | Multiple exon skipping compositions for dmd |
HUE040445T2 (en) | 2009-11-12 | 2019-03-28 | Univ Western Australia | Antisense molecules and methods for treating pathologies |
BR122020016865B1 (en) | 2013-03-14 | 2022-12-27 | Sarepta Therapeutics, Inc. | ANTISENSE OLIGONUCLEOTIDE, PHARMACEUTICAL COMPOSITION INCLUDING THE SAME AND USE OF SAID COMPOSITION FOR THE TREATMENT OF DUCHENNE MUSCULAR DYSTROPHY (DMD) |
CA2906812A1 (en) | 2013-03-15 | 2014-09-18 | Sarepta Therapeutics, Inc. | Improved compositions for treating muscular dystrophy |
UA123359C2 (en) * | 2015-09-15 | 2021-03-24 | Ніппон Шин'Яку Ко., Лтд. | Antisense nucleic acid |
Citations (3)
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) |
-
2009
- 2009-06-26 IT IT000487A patent/ITTO20090487A1/en unknown
-
2010
- 2010-06-25 WO PCT/IB2010/052912 patent/WO2010150231A1/en active Application Filing
Patent Citations (3)
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)
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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