DE60330136D1 - Chimäre snrna moleküle mit antisense-sequenzen gegen die "splice junctions" des dystrophin-gens und ihre therapeutischen verwendungen - Google Patents
Chimäre snrna moleküle mit antisense-sequenzen gegen die "splice junctions" des dystrophin-gens und ihre therapeutischen verwendungenInfo
- Publication number
- DE60330136D1 DE60330136D1 DE60330136T DE60330136T DE60330136D1 DE 60330136 D1 DE60330136 D1 DE 60330136D1 DE 60330136 T DE60330136 T DE 60330136T DE 60330136 T DE60330136 T DE 60330136T DE 60330136 D1 DE60330136 D1 DE 60330136D1
- Authority
- DE
- Germany
- Prior art keywords
- gene
- splice junctions
- dystrophine
- therapeutic uses
- antisense sequences
- 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.)
- Expired - Lifetime
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
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)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Preparation Of Compounds By Using Micro-Organisms (AREA)
- Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
Applications Claiming Priority (2)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
IT2002RM000253A ITRM20020253A1 (it) | 2002-05-08 | 2002-05-08 | Molecole chimeriche di snrna con sequenze antisenso per le giunzioni di splicing del gene della distrofina e applicazioni terapeutiche. |
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 |
Publications (1)
Publication Number | Publication Date |
---|---|
DE60330136D1 true DE60330136D1 (de) | 2009-12-31 |
Family
ID=11456291
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
DE60330136T Expired - Lifetime DE60330136D1 (de) | 2002-05-08 | 2003-05-06 | Chimäre snrna moleküle mit antisense-sequenzen gegen die "splice junctions" des dystrophin-gens und ihre therapeutischen verwendungen |
Country Status (11)
Country | Link |
---|---|
EP (2) | EP1501931B1 (de) |
AT (1) | ATE449170T1 (de) |
AU (1) | AU2003231928A1 (de) |
CY (1) | CY1109746T1 (de) |
DE (1) | DE60330136D1 (de) |
DK (1) | DK1501931T3 (de) |
ES (1) | ES2336909T3 (de) |
IT (1) | ITRM20020253A1 (de) |
PT (1) | PT1501931E (de) |
SI (1) | SI1501931T1 (de) |
WO (1) | WO2003095647A2 (de) |
Families Citing this family (51)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
AUPP249298A0 (en) | 1998-03-20 | 1998-04-23 | Ag-Gene Australia Limited | Synthetic genes and genetic constructs comprising same I |
SK287538B6 (sk) | 1998-03-20 | 2011-01-04 | Commonwealth Scientific And Industrial Research Organisation | Syntetický gén obsahujúci dispergovanú alebo cudzorodú deoxyribonukleovú molekulu, génový konštrukt obsahujúci tento syntetický gén a prípravok obsahujúci tento syntetický gén |
US6423885B1 (en) | 1999-08-13 | 2002-07-23 | Commonwealth Scientific And Industrial Research Organization (Csiro) | Methods for obtaining modified phenotypes in plant cells |
AUPR621501A0 (en) | 2001-07-06 | 2001-08-02 | Commonwealth Scientific And Industrial Research Organisation | Delivery of ds rna |
CA2568603A1 (en) * | 2003-09-12 | 2005-03-24 | Commonwealth Scientific And Industrial Research Organisation | Modified gene-silencing nucleic acid molecules and uses thereof |
FR2874384B1 (fr) | 2004-08-17 | 2010-07-30 | Genethon | Vecteur viral adeno-associe pour realiser du saut d'exons dans un gene codant une proteine a domaines dispensables |
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 (de) | 2006-05-19 | 2007-11-21 | Academisch Ziekenhuis Leiden | Mittel und Verfahren zur Induktion von Exon-skipping |
DK2049664T3 (da) | 2006-08-11 | 2012-01-02 | Prosensa Technologies Bv | Enkeltstrengede oligonukleotider, som er komplementære til repetitive elementer, til behandling af med DNA-repetitiv-ustabilitet associerede lidelser |
WO2008113773A2 (en) * | 2007-03-16 | 2008-09-25 | Biorigen S.R.L. | Gene expression regulation technology and noncoding rnas for diagnosis and therapy |
US9441221B2 (en) | 2007-03-30 | 2016-09-13 | Rutgers, The State University Of New Jersey | Compositions and methods for gene silencing |
US8907075B2 (en) | 2007-03-30 | 2014-12-09 | Samuel Ian Gunderson | 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 |
EP2142672B1 (de) * | 2007-03-30 | 2012-09-05 | Rutgers, The State University of New Jersey | Zusammensetzungen und Verfahren zum Gen-Silencing |
JP2010533170A (ja) | 2007-07-12 | 2010-10-21 | プロセンサ テクノロジーズ ビー.ブイ. | 化合物を種々の選択された臓器、組織又は腫瘍細胞に標的化するための分子 |
JP5706157B2 (ja) | 2007-07-12 | 2015-04-22 | プロセンサ テクノロジーズ ビー.ブイ.Prosensa Technologies B.V. | 化合物を種々の選択臓器又は組織に標的化するための分子 |
EP2203173B1 (de) | 2007-10-26 | 2015-12-23 | Academisch Ziekenhuis Leiden | Mittel und verfahren zur bekämpfung von muskelleiden |
USRE48468E1 (en) | 2007-10-26 | 2021-03-16 | Biomarin Technologies B.V. | Means and methods for counteracting muscle disorders |
WO2009099326A1 (en) | 2008-02-08 | 2009-08-13 | Prosensa Holding Bv | Methods and means for treating dna repeat instability associated genetic disorders |
EP2119783A1 (de) | 2008-05-14 | 2009-11-18 | Prosensa Technologies B.V. | Verfahren für effizientes Exon (44)-Skipping bei Duchenne-Muskeldystrophie und entsprechende Mittel |
US8889641B2 (en) | 2009-02-11 | 2014-11-18 | The University Of North Carolina At Chapel Hill | Modified virus vectors and methods of making and using the same |
US20120046342A1 (en) | 2009-04-24 | 2012-02-23 | Prosensa Technologies B.V. | Oligonucleotide comprising an inosine for treating dmd |
EP2258863A1 (de) * | 2009-05-25 | 2010-12-08 | Universita'Degli Studi di Roma "La Sapienza" | miRNA biomarkers für die Diagnose und Weiterentwicklung der Duchenne Dystrophie, für die Überwachung therapeutischer Interventionen und als Therapeutik |
JP6141018B2 (ja) | 2009-12-24 | 2017-06-07 | バイオマリン テクノロジーズ ベー.フェー. | 炎症性障害を治療するための分子 |
EP2524037B1 (de) | 2010-01-12 | 2018-05-16 | The University Of North Carolina At Chapel Hill | Restriktive umgekehrte endverstärkungen für virusvektoren |
TWI541024B (zh) | 2010-09-01 | 2016-07-11 | 日本新藥股份有限公司 | 反義核酸 |
DK2673289T3 (da) | 2011-02-10 | 2023-07-24 | Univ North Carolina Chapel Hill | Virusvektorer med modificerede transduktionsprofiler og fremgangsmåder til fremstilling og anvendelse deraf |
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 |
CN104203289B (zh) | 2012-01-27 | 2020-11-03 | 比奥马林技术公司 | 用于治疗杜兴型肌营养不良症和贝克型肌营养不良症的具有改善特性的rna调节性寡核苷酸 |
AU2013323270B2 (en) | 2012-09-28 | 2017-09-28 | The University Of North Carolina At Chapel Hill | AAV vectors targeted to oligodendrocytes |
AU2014227766B2 (en) | 2013-03-15 | 2018-10-04 | The University Of North Carolina At Chapel Hill | Methods and compositions for dual glycan binding AAV vectors |
EP2970966A4 (de) | 2013-03-15 | 2016-10-26 | Univ North Carolina | Synthetische adenoassoziierte virusinvertierte endwiederholungssequenzen |
CA2967393A1 (en) | 2014-11-21 | 2016-05-26 | The University Of North Carolina At Chapel Hill | Aav vectors targeted to the central nervous system |
CA3221229A1 (en) | 2015-01-14 | 2016-07-21 | The University Of North Carolina At Chapel Hill | Methods and compositions for targeted gene transfer |
CA2977071A1 (en) | 2015-02-20 | 2016-08-25 | Board Of Regents, The University Of Texas System | Methods and compositions for attenuated chlamydia as vaccine and vector |
AU2016332821B2 (en) | 2015-09-28 | 2022-02-17 | The University Of North Carolina At Chapel Hill | Methods and compositions for antibody-evading virus vectors |
CA3054711A1 (en) | 2017-03-15 | 2018-09-20 | The University Of North Carolina At Chapel Hill | Polyploid adeno-associated virus vectors and methods of making and using the same |
EP3774854A1 (de) | 2018-04-03 | 2021-02-17 | Stridebio, Inc. | Antikörperausweichende virusvektoren |
AU2019247746A1 (en) | 2018-04-03 | 2020-10-15 | Ginkgo Bioworks, Inc. | Antibody-evading virus vectors |
EA202092362A1 (ru) | 2018-04-03 | 2021-01-11 | Страйдбайо, Инк. | Вирусные векторы для нацеливания на ткани глаза |
JP2021528959A (ja) | 2018-06-22 | 2021-10-28 | アスクレピオス バイオファーマシューティカル, インコーポレイテッド | 細胞内で存続する遺伝子送達のためのベクター |
EA202192501A1 (ru) | 2019-03-21 | 2021-12-16 | Страйдбайо, Инк. | Рекомбинантные аденоассоциированные вирусные векторы |
JP2022529662A (ja) | 2019-04-26 | 2022-06-23 | ザ・ユニヴァーシティ・オヴ・ノース・キャロライナ・アト・チャペル・ヒル | 二重グリカン結合aav2.5ベクターのための方法および組成物 |
US11905523B2 (en) | 2019-10-17 | 2024-02-20 | Ginkgo Bioworks, Inc. | Adeno-associated viral vectors for treatment of Niemann-Pick Disease type-C |
CA3196036A1 (en) | 2020-10-28 | 2022-05-05 | The University Of North Carolina At Chapel Hill | Methods and compositions for dual glycan binding aav2.5 vector |
WO2022093769A1 (en) | 2020-10-28 | 2022-05-05 | The University Of North Carolina At Chapel Hill | Methods and compositions for dual glycan binding aav2.5 vector |
TW202242124A (zh) | 2021-01-14 | 2022-11-01 | 美商史崔德生物公司 | 靶向t細胞之aav載體 |
JP2024503725A (ja) | 2021-01-15 | 2024-01-26 | アウトペース バイオ インコーポレイテッド | 小分子調節遺伝子発現システム |
TW202342759A (zh) | 2022-02-04 | 2023-11-01 | 美商史崔德生物公司 | 重組腺相關病毒載體及其使用方法 |
US20230348909A1 (en) * | 2022-03-30 | 2023-11-02 | Biomarin Pharmaceutical Inc. | Dystrophin exon skipping oligonucleotides |
CN115806984B (zh) * | 2022-10-18 | 2023-10-10 | 昆明理工大学 | 环状rna及载体和载体的应用 |
Family Cites Families (1)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
EP1191097A1 (de) * | 2000-09-21 | 2002-03-27 | Leids Universitair Medisch Centrum | Induktion von "exon-skipping" in eukaryotischen Zellen |
-
2002
- 2002-05-08 IT IT2002RM000253A patent/ITRM20020253A1/it unknown
-
2003
- 2003-05-06 EP EP03749984A patent/EP1501931B1/de not_active Expired - Lifetime
- 2003-05-06 DK DK03749984.5T patent/DK1501931T3/da active
- 2003-05-06 AT AT03749984T patent/ATE449170T1/de active
- 2003-05-06 WO PCT/IT2003/000273 patent/WO2003095647A2/en not_active Application Discontinuation
- 2003-05-06 PT PT03749984T patent/PT1501931E/pt unknown
- 2003-05-06 ES ES03749984T patent/ES2336909T3/es not_active Expired - Lifetime
- 2003-05-06 EP EP09176221.1A patent/EP2151497B1/de not_active Expired - Lifetime
- 2003-05-06 AU AU2003231928A patent/AU2003231928A1/en not_active Abandoned
- 2003-05-06 DE DE60330136T patent/DE60330136D1/de not_active Expired - Lifetime
- 2003-05-06 SI SI200331749T patent/SI1501931T1/sl unknown
-
2010
- 2010-02-03 CY CY20101100102T patent/CY1109746T1/el unknown
Also Published As
Publication number | Publication date |
---|---|
ATE449170T1 (de) | 2009-12-15 |
EP2151497A2 (de) | 2010-02-10 |
AU2003231928A1 (en) | 2003-11-11 |
WO2003095647A3 (en) | 2004-02-19 |
EP1501931B1 (de) | 2009-11-18 |
WO2003095647A2 (en) | 2003-11-20 |
SI1501931T1 (sl) | 2010-03-31 |
EP2151497B1 (de) | 2014-01-08 |
ITRM20020253A0 (it) | 2002-05-08 |
PT1501931E (pt) | 2010-02-23 |
EP2151497A3 (de) | 2010-03-10 |
EP1501931A2 (de) | 2005-02-02 |
CY1109746T1 (el) | 2014-09-10 |
ES2336909T3 (es) | 2010-04-19 |
ITRM20020253A1 (it) | 2003-11-10 |
DK1501931T3 (da) | 2010-03-29 |
AU2003231928A8 (en) | 2003-11-11 |
Similar Documents
Publication | Publication Date | Title |
---|---|---|
DE60330136D1 (de) | Chimäre snrna moleküle mit antisense-sequenzen gegen die "splice junctions" des dystrophin-gens und ihre therapeutischen verwendungen | |
CY1116385T1 (el) | Αντινοηματικη διαμορφωση της εκφρασης της απολιποπρωτεινης β | |
DE60135936D1 (de) | Induktion von "exon-skipping" in eukaryotischen Zellen | |
DE60327786D1 (de) | Kdr-peptide und diese enthaltende impfstoffe | |
ITRM20030149A1 (it) | Oligonucleotidi (odn) antisenso per smad7 e loro usi in campo medico | |
EP2253706A3 (de) | Antisense-Modulation von Kinesin-1-Expression | |
EA200300929A1 (ru) | Новые лекарственные композиции на основе антихолинергических средств и ингибиторов pde-iv | |
WO2004098377A3 (en) | Methods and compositions for diagnosis and therapy of parkin-associated disorders | |
WO2003054154A3 (en) | Antisense modulation of mucin 1, transmembrane expression | |
EA200400812A1 (ru) | Секретируемый белок | |
WO2003012031A3 (en) | Antisense modulation of stearoyl-coa desaturase expression | |
EP1506216A4 (de) | Antisense-modulierung der kinesin-like-1-expression | |
IL169256A0 (en) | Metalloprotease proteins | |
ATE492528T1 (de) | Derivate und pharmazeutische zusammensetzungen von n-hydroxyalkyltetramethyl cyclo propan carboxamid mit antiepileptischer und verfahren zu deren herstellung | |
NO20052525D0 (no) | Spleisevariant av humant hypofyseveksthormon | |
WO2003040161A3 (en) | Antisense modulation of activating transcription factor 3 expression | |
WO2004001061A3 (en) | Antisense modulation of heme oxygenase 1 expression | |
AU2003255504A8 (en) | Use of 6 amino-quinoline-5, 8 quinones and nucleic acids associated with senescence for the treatment of tumors | |
WO2003040320A3 (en) | Antisense modulation of human collapsin response mediator protein 2 expression | |
WO2003106647A3 (en) | ANTISENSE MODULATION OF JUNCTION ADHESION MOLECULE 3 EXPRESSION | |
CY1114199T1 (el) | Αντιπληροφοριακη διαμορφωση της εκφρασης της απολιποπρωτεϊνης β | |
WO2003051309A3 (en) | Antisense modulation of abc transporter mhc 1 expression | |
WO2002095022A3 (en) | Sequence-specific dna binding compounds and their use in targeting telomeres and other dna sequences | |
WO2002062845A3 (en) | Adhesion molecules | |
EA200501843A1 (ru) | Tnf-подобный секретируемый белок |
Legal Events
Date | Code | Title | Description |
---|---|---|---|
8364 | No opposition during term of opposition |