WO2003095647A3 - CHIMERIC snRNA MOLECULES CARRYING ANTISENSE SEQUENCES AGAINST THE SPLICE JUNCTIONS OF THE DYSTROPHIN GENE AND THEIR THERAPEUTIC APPLICATIONS - Google Patents
CHIMERIC snRNA MOLECULES CARRYING ANTISENSE SEQUENCES AGAINST THE SPLICE JUNCTIONS OF THE DYSTROPHIN GENE AND THEIR THERAPEUTIC APPLICATIONS Download PDFInfo
- Publication number
- WO2003095647A3 WO2003095647A3 PCT/IT2003/000273 IT0300273W WO03095647A3 WO 2003095647 A3 WO2003095647 A3 WO 2003095647A3 IT 0300273 W IT0300273 W IT 0300273W WO 03095647 A3 WO03095647 A3 WO 03095647A3
- Authority
- WO
- WIPO (PCT)
- Prior art keywords
- splice junctions
- gene
- therapeutic applications
- antisense sequences
- dystrophin gene
- 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
-
- 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
- C12N2310/11—Antisense
- C12N2310/111—Antisense spanning the whole gene, or a large part of it
Abstract
Priority Applications (6)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
AU2003231928A AU2003231928A1 (en) | 2002-05-08 | 2003-05-06 | CHIMERIC snRNA MOLECULES CARRYING ANTISENSE SEQUENCES AGAINST THE SPLICE JUNCTIONS OF THE DYSTROPHIN GENE AND THEIR THERAPEUTIC APPLICATIONS |
DE60330136T DE60330136D1 (en) | 2002-05-08 | 2003-05-06 | CHIMERIC SNRNA MOLECULES WITH ANTISENSE SEQUENCES AGAINST THE "SPLICE JUNCTIONS" OF THE DYSTROPHINE GENE AND THEIR THERAPEUTIC USES |
AT03749984T ATE449170T1 (en) | 2002-05-08 | 2003-05-06 | CHIMERIC SNRNA MOLECULES WITH ANTISENSE SEQUENCES AGAINST THE ßSPLICE JUNCTIONSß OF THE DYSTROPHIN GENE AND THEIR THERAPEUTIC USES |
EP03749984A EP1501931B1 (en) | 2002-05-08 | 2003-05-06 | Chimeric snrna molecules carrying antisense sequences against the splice junctions of the dystrophin gene and their therapeutic applications |
DK03749984.5T DK1501931T3 (en) | 2002-05-08 | 2003-05-06 | Chimeric snRNA molecules that carry antisense sequences against the splicing compounds of the dystrophin gene and their therapeutic applications |
SI200331749T SI1501931T1 (en) | 2002-05-08 | 2003-05-06 | Chimeric snrna molecules carrying antisense sequences against the splice junctions of the dystrophin gene and their therapeutic applications |
Applications Claiming Priority (2)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
ITRM2002A000253 | 2002-05-08 | ||
IT2002RM000253A ITRM20020253A1 (en) | 2002-05-08 | 2002-05-08 | SNRNA CHEMICAL MOLECULES WITH ANTISENSE SEQUENCES FOR SPLICING JUNCTIONS OF THE DYSTROPHINE GENE AND THERAPEUTIC APPLICATIONS. |
Publications (2)
Publication Number | Publication Date |
---|---|
WO2003095647A2 WO2003095647A2 (en) | 2003-11-20 |
WO2003095647A3 true WO2003095647A3 (en) | 2004-02-19 |
Family
ID=11456291
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
PCT/IT2003/000273 WO2003095647A2 (en) | 2002-05-08 | 2003-05-06 | CHIMERIC snRNA MOLECULES CARRYING ANTISENSE SEQUENCES AGAINST THE SPLICE JUNCTIONS OF THE DYSTROPHIN GENE AND THEIR THERAPEUTIC APPLICATIONS |
Country Status (11)
Country | Link |
---|---|
EP (2) | EP2151497B1 (en) |
AT (1) | ATE449170T1 (en) |
AU (1) | AU2003231928A1 (en) |
CY (1) | CY1109746T1 (en) |
DE (1) | DE60330136D1 (en) |
DK (1) | DK1501931T3 (en) |
ES (1) | ES2336909T3 (en) |
IT (1) | ITRM20020253A1 (en) |
PT (1) | PT1501931E (en) |
SI (1) | SI1501931T1 (en) |
WO (1) | WO2003095647A2 (en) |
Cited By (2)
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---|---|---|---|---|
US9139828B2 (en) | 2008-05-14 | 2015-09-22 | Prosensa Technologies B.V. | Method for efficient exon (44) skipping in duchenne muscular dystrophy and associated means |
US9243245B2 (en) | 2007-10-26 | 2016-01-26 | Academisch Ziekenhuis Leiden | Means and methods for counteracting muscle disorders |
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AUPP249298A0 (en) | 1998-03-20 | 1998-04-23 | Ag-Gene Australia Limited | Synthetic genes and genetic constructs comprising same I |
EP1555317B1 (en) | 1998-03-20 | 2011-09-28 | Commonwealth Scientific And Industrial Research Organisation | Synthetic genes and genetic constructs comprising the same |
US6423885B1 (en) | 1999-08-13 | 2002-07-23 | Commonwealth Scientific And Industrial Research Organization (Csiro) | Methods for obtaining modified phenotypes in plant cells |
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WO2005026356A1 (en) * | 2003-09-12 | 2005-03-24 | Commonwealth Scientific And Industrial Research Organisation | Modified gene-silencing nucleic acid molecules and uses thereof |
FR2874384B1 (en) | 2004-08-17 | 2010-07-30 | Genethon | ADENO-ASSOCIATED VIRAL VECTOR FOR PRODUCING EXON JUMP IN A GENE ENCODING A PROTEIN WITH DISPENSABLE DOMAINS |
WO2007123391A1 (en) | 2006-04-20 | 2007-11-01 | Academisch Ziekenhuis Leiden | Therapeutic intervention in a genetic disease in an individual by modifying expression of an aberrantly expressed gene. |
EP1857548A1 (en) | 2006-05-19 | 2007-11-21 | Academisch Ziekenhuis Leiden | Means and method for inducing exon-skipping |
DK2049664T3 (en) | 2006-08-11 | 2012-01-02 | Prosensa Technologies Bv | Single-stranded oligonucleotides, complementary to repetitive elements, for the treatment of DNA repetitive instability-associated disorders |
EP2152873A2 (en) * | 2007-03-16 | 2010-02-17 | Biorigen S.r.l | Gene expression regulation technology and noncoding rnas for diagnosis and therapy |
US8907075B2 (en) | 2007-03-30 | 2014-12-09 | Samuel Ian Gunderson | Compositions and methods for gene silencing |
WO2008121963A2 (en) * | 2007-03-30 | 2008-10-09 | Rutgers, The State University Of New Jersey | Compositions and methods for gene silencing |
US8343941B2 (en) | 2007-03-30 | 2013-01-01 | Rutgers, The State University Of New Jersey | Compositions and methods for gene silencing |
US9441221B2 (en) | 2007-03-30 | 2016-09-13 | Rutgers, The State University Of New Jersey | Compositions and methods for gene silencing |
WO2009008727A2 (en) | 2007-07-12 | 2009-01-15 | Prosensa Technologies B.V. | Molecules for targeting compounds to various selected organs or tissues |
WO2009008725A2 (en) | 2007-07-12 | 2009-01-15 | Prosensa Technologies B.V. | Molecules for targeting compounds to various selected organs, tissues or tumor cells |
USRE48468E1 (en) | 2007-10-26 | 2021-03-16 | Biomarin Technologies B.V. | Means and methods for counteracting muscle disorders |
NZ587178A (en) | 2008-02-08 | 2011-11-25 | Prosensa Holding Bv | Methods and means for treating dna repeat instability associated genetic disorders |
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AU2010239779A1 (en) | 2009-04-24 | 2011-11-17 | Prosensa Technologies B.V. | Oligonucleotide comprising an inosine for treating DMD |
EP2258863A1 (en) * | 2009-05-25 | 2010-12-08 | Universita'Degli Studi di Roma "La Sapienza" | miRNA biomarkers for the diagnosis of duchenne muscular dystrophy progression, for monitoring therapeutic interventions, and as therapeutics |
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US9303258B2 (en) | 2011-04-05 | 2016-04-05 | The Regents Of The University Of California | Method and compositions comprising small RNA agonist and antagonists to modulate inflammation |
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TW202242124A (en) | 2021-01-14 | 2022-11-01 | 美商史崔德生物公司 | Aav vectors targeting t-cells |
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TW202342759A (en) | 2022-02-04 | 2023-11-01 | 美商史崔德生物公司 | Recombinant adeno-associated virus vectors, and methods of use thereof |
TW202342070A (en) * | 2022-03-30 | 2023-11-01 | 美商拜奧馬林製藥公司 | Dystrophin exon skipping oligonucleotides |
CN115806984B (en) * | 2022-10-18 | 2023-10-10 | 昆明理工大学 | Circular RNA and vector and application of vector |
Citations (1)
Publication number | Priority date | Publication date | Assignee | Title |
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EP1191097A1 (en) * | 2000-09-21 | 2002-03-27 | Leids Universitair Medisch Centrum | Induction of exon skipping in eukaryotic cells |
-
2002
- 2002-05-08 IT IT2002RM000253A patent/ITRM20020253A1/en unknown
-
2003
- 2003-05-06 PT PT03749984T patent/PT1501931E/en unknown
- 2003-05-06 EP EP09176221.1A patent/EP2151497B1/en not_active Expired - Lifetime
- 2003-05-06 DE DE60330136T patent/DE60330136D1/en not_active Expired - Lifetime
- 2003-05-06 DK DK03749984.5T patent/DK1501931T3/en active
- 2003-05-06 WO PCT/IT2003/000273 patent/WO2003095647A2/en not_active Application Discontinuation
- 2003-05-06 AT AT03749984T patent/ATE449170T1/en active
- 2003-05-06 AU AU2003231928A patent/AU2003231928A1/en not_active Abandoned
- 2003-05-06 EP EP03749984A patent/EP1501931B1/en not_active Expired - Lifetime
- 2003-05-06 ES ES03749984T patent/ES2336909T3/en not_active Expired - Lifetime
- 2003-05-06 SI SI200331749T patent/SI1501931T1/en unknown
-
2010
- 2010-02-03 CY CY20101100102T patent/CY1109746T1/en unknown
Patent Citations (1)
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EP1191097A1 (en) * | 2000-09-21 | 2002-03-27 | Leids Universitair Medisch Centrum | Induction of exon skipping in eukaryotic cells |
Non-Patent Citations (7)
Title |
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BRUN C ET AL: "U7 snRNAs induce correction of mutated dystrophin pre-mRNA by exon skipping.", CMLS CELLULAR AND MOLECULAR LIFE SCIENCES, vol. 60, no. 3, March 2003 (2003-03-01), pages 557 - 566, XP009021235, ISSN: 1420-682X * |
DE ANGELIS FERNANDA GABRIELLA ET AL: "Chimeric snRNA molecules carrying antisense sequences against the splice junctions of exon 51 of the dystrophin pre-mRNA induce exon skipping and restoration of a dystrophin synthesis in Delta 48-50 DMD cells.", PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA. UNITED STATES 9 JUL 2002, vol. 99, no. 14, 9 July 2002 (2002-07-09), pages 9456 - 9461, XP002261982, ISSN: 0027-8424 * |
DEUTEKOM VAN J C T ET AL: "Antisense-induced exon skipping restores dystrophin expression in DMD patient derived muscle cells", HUMAN MOLECULAR GENETICS, OXFORD UNIVERSITY PRESS, SURREY, GB, vol. 10, no. 15, 2001, pages 1547 - 1554, XP002250908, ISSN: 0964-6906 * |
GORMAN ET AL.: "Restoration of Correct Splicing of Thalassemic ß-Globin Pre-mRNA by modified U1 snRNAs", J BIOL CHEM, vol. 275, no. 46, 17 November 2000 (2000-11-17), pages 35914 - 35919, XP002261981 * |
M. MATSUO: "Duchenne and Becker Muscular Dystrophy: From Gene Diagnosis to Molecular Therapy", IUBMB LIFE, vol. 53, no. 3, March 2002 (2002-03-01), pages 147 - 152, XP009021242 * |
MANN C J ET AL: "ANTISENSE-INDUCED EXON SKIPPING AND SYNTHESIS OF DYSTROPHIN IN THE MDX MOUSE", PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF USA, NATIONAL ACADEMY OF SCIENCE. WASHINGTON, US, vol. 98, no. 1, 2 January 2001 (2001-01-02), pages 42 - 47, XP001160821, ISSN: 0027-8424 * |
SUTER D ET AL: "DOUBLE-TARGET ANTISENSE U7 SNRNAS PROMOTE EFFICIENT SKIPPING OF AN ABERRANT EXON IN THREE HUMAN BETA-THALASSEMIC MUTATIONS", HUMAN MOLECULAR GENETICS, OXFORD UNIVERSITY PRESS, SURREY, GB, vol. 8, December 1999 (1999-12-01), pages 2415 - 2423, XP000973208, ISSN: 0964-6906 * |
Cited By (4)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
US9243245B2 (en) | 2007-10-26 | 2016-01-26 | Academisch Ziekenhuis Leiden | Means and methods for counteracting muscle disorders |
US9499818B2 (en) | 2007-10-26 | 2016-11-22 | BioMarin Technologies, B.V. | Methods and means for efficient skipping of at least one of the exons 51-53, 55, 57 and 59 of the human duchenne muscular dystrophy gene |
US9528109B2 (en) | 2007-10-26 | 2016-12-27 | Biomarin Technologies B.V. | Methods and means for efficient skipping of exon 45 in duchenne muscular dystrophy pre-mRNA |
US9139828B2 (en) | 2008-05-14 | 2015-09-22 | Prosensa Technologies B.V. | Method for efficient exon (44) skipping in duchenne muscular dystrophy and associated means |
Also Published As
Publication number | Publication date |
---|---|
AU2003231928A8 (en) | 2003-11-11 |
DK1501931T3 (en) | 2010-03-29 |
DE60330136D1 (en) | 2009-12-31 |
EP2151497A3 (en) | 2010-03-10 |
ITRM20020253A1 (en) | 2003-11-10 |
EP1501931B1 (en) | 2009-11-18 |
EP2151497B1 (en) | 2014-01-08 |
ATE449170T1 (en) | 2009-12-15 |
PT1501931E (en) | 2010-02-23 |
ES2336909T3 (en) | 2010-04-19 |
ITRM20020253A0 (en) | 2002-05-08 |
WO2003095647A2 (en) | 2003-11-20 |
EP2151497A2 (en) | 2010-02-10 |
SI1501931T1 (en) | 2010-03-31 |
CY1109746T1 (en) | 2014-09-10 |
EP1501931A2 (en) | 2005-02-02 |
AU2003231928A1 (en) | 2003-11-11 |
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