ITTO20090487A1 - Oligonucleotidi antisenso atti ad indurre lo skipping esonico e loro impiego come medicamento per il trattamento della distrofia muscolare di duchenne (dmd) - Google Patents

Oligonucleotidi antisenso atti ad indurre lo skipping esonico e loro impiego come medicamento per il trattamento della distrofia muscolare di duchenne (dmd)

Info

Publication number: ITTO20090487A1
Authority: IT; Italy
Prior art keywords: indocate; exonic; dmd; skipping; medication
Prior art date: 2009-06-26

Application number

IT000487A

Other languages

English (en)

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.)

2009-06-26

Filing date

2009-06-26

Publication date

2010-12-27

2009-06-26 Application filed by Univ Ferrara filed Critical Univ Ferrara

2009-06-26 Priority to IT000487A priority Critical patent/ITTO20090487A1/it

2010-06-25 Priority to PCT/IB2010/052912 priority patent/WO2010150231A1/en

2010-12-27 Publication of ITTO20090487A1 publication Critical patent/ITTO20090487A1/it

Links

206010013801 Duchenne Muscular Dystrophy Diseases 0.000 title 2
YKWQHMLRAPIWDP-UHFFFAOYSA-N 2-(dimethylamino)ethyl 1-benzyl-2,3-dimethylindole-5-carboxylate Chemical compound CC1=C(C)C2=CC(C(=O)OCCN(C)C)=CC=C2N1CC1=CC=CC=C1 YKWQHMLRAPIWDP-UHFFFAOYSA-N 0.000 title 1
230000000692 anti-sense effect Effects 0.000 title 1
229940079593 drug Drugs 0.000 title 1
239000003814 drug Substances 0.000 title 1
229950001049 indocate Drugs 0.000 title 1

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 Oligonucleotidi antisenso atti ad indurre lo skipping esonico e loro impiego come medicamento per il trattamento della distrofia muscolare di duchenne (dmd) ITTO20090487A1 (it)

Priority Applications (2)

Application Number	Priority Date	Filing Date	Title
IT000487A ITTO20090487A1 (it)	2009-06-26	2009-06-26	Oligonucleotidi antisenso atti ad indurre lo skipping esonico e loro impiego come medicamento per il trattamento della distrofia muscolare di duchenne (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 (it)	2009-06-26	2009-06-26	Oligonucleotidi antisenso atti ad indurre lo skipping esonico e loro impiego come medicamento per il trattamento della distrofia muscolare di duchenne (dmd)

Publications (1)

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

Family

ID=41508769

Family Applications (1)

Application Number	Title	Priority Date	Filing Date
IT000487A ITTO20090487A1 (it)	2009-06-26	2009-06-26	Oligonucleotidi antisenso atti ad indurre lo skipping esonico e loro impiego come medicamento per il trattamento della distrofia muscolare di duchenne (dmd)

Country Status (2)

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

Families Citing this family (7)

* Cited by examiner, † Cited by third party
Publication number	Priority date	Publication date	Assignee	Title
EP2933332A1 (en)	2004-06-28	2015-10-21	The 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
BR122020021379B1 (pt)	2008-10-24	2021-05-11	Sarepta Therapeutics, Inc.	oligômero morfolino fosforodiamidato, composição que compreende o mesmo e uso do dito oligômero para tratar distrofia muscular
TR201816523T4 (tr)	2009-11-12	2018-11-21	Univ Western Australia	Patolojilerin tedavisine yönelik antisens moleküller ve yöntemler.
CN110218727A (zh)	2013-03-14	2019-09-10	萨勒普塔医疗公司	用于治疗肌营养不良的外显子跳跃组合物
US20140329762A1 (en)	2013-03-15	2014-11-06	Sarepta Therapeutics, Inc.	Compositions for treating muscular dystrophy
HUE050061T2 (hu) *	2015-09-15	2020-12-28	Nippon Shinyaku Co Ltd	Antiszensz nukleinsav

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)

2009
- 2009-06-26 IT IT000487A patent/ITTO20090487A1/it unknown
2010
- 2010-06-25 WO PCT/IB2010/052912 patent/WO2010150231A1/en not_active Ceased

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] *

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

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