ITRM20020253A0 - Molecole chimeriche di snrna con sequenze antisenso per le giunzioni di splicing del gene della distrofina e applicazioni terapeutiche. - Google Patents
Molecole chimeriche di snrna con sequenze antisenso per le giunzioni di splicing del gene della distrofina e applicazioni terapeutiche.Info
- Publication number
- ITRM20020253A0 ITRM20020253A0 IT2002RM000253A ITRM20020253A ITRM20020253A0 IT RM20020253 A0 ITRM20020253 A0 IT RM20020253A0 IT 2002RM000253 A IT2002RM000253 A IT 2002RM000253A IT RM20020253 A ITRM20020253 A IT RM20020253A IT RM20020253 A0 ITRM20020253 A0 IT RM20020253A0
- Authority
- IT
- Italy
- Prior art keywords
- dytrophin
- therapeutic applications
- antisense sequences
- gene splicing
- splicing junctions
- Prior art date
Links
- 230000000692 anti-sense effect Effects 0.000 title 1
- 108090000623 proteins and genes Proteins 0.000 title 1
- 108091029842 small nuclear ribonucleic acid Proteins 0.000 title 1
- 230000001225 therapeutic effect Effects 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
-
- 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)
Priority Applications (12)
| 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. |
| DE60330136T 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 |
| DK03749984.5T DK1501931T3 (da) | 2002-05-08 | 2003-05-06 | Kimære snRNA-molekyler, som bærer antisense-sekvenser mod splejsningsforbindelserne af dystrophingenet, og deres terapeutiske anvendelser |
| SI200331749T SI1501931T1 (sl) | 2002-05-08 | 2003-05-06 | Himerne molekule snrna ki nosijo protismiselne sekvence proti mestu cepitve distrofinskega gena innjihove terapevtske aplikacije |
| EP09176221.1A EP2151497B1 (en) | 2002-05-08 | 2003-05-06 | Chimeric U1 and U2 snRNA molecules carrying antisense sequences against the splice junctions of the dystrophin gene and their therapeutic applications |
| PT03749984T PT1501931E (pt) | 2002-05-08 | 2003-05-06 | Moléculas de arnnp (snrna) quiméricas portadoras de sequências anti-sentido contra as junções de splicing do gene da distrofina e suas aplicações terapêuticas |
| 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 |
| 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 |
| AT03749984T ATE449170T1 (de) | 2002-05-08 | 2003-05-06 | CHIMÄRE SNRNA MOLEKÜLE MIT ANTISENSE-SEQUENZEN GEGEN DIE ßSPLICE JUNCTIONSß DES DYSTROPHIN-GENS UND IHRE THERAPEUTISCHEN VERWENDUNGEN |
| ES03749984T ES2336909T3 (es) | 2002-05-08 | 2003-05-06 | Molaculas de arnsn quimericas que transportan secuencias antisentido contra las uniones por corte y empalme del gen de la distrofina y sus aplicaciones terapeuticas. |
| 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 |
| CY20101100102T CY1109746T1 (el) | 2002-05-08 | 2010-02-03 | Χιμαιρικα μορια snrna που φερουν αντιπληροφοριακες αλληλουχιες εναντι των συνδεσμων συρραφης του γονιδιου δυστροφινης και θεραπευτικες εφαρμογες αυτων |
Applications Claiming Priority (1)
| 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. |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| ITRM20020253A0 true ITRM20020253A0 (it) | 2002-05-08 |
| ITRM20020253A1 ITRM20020253A1 (it) | 2003-11-10 |
Family
ID=11456291
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| 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. |
Country Status (11)
| Country | Link |
|---|---|
| EP (2) | EP1501931B1 (it) |
| AT (1) | ATE449170T1 (it) |
| AU (1) | AU2003231928A1 (it) |
| CY (1) | CY1109746T1 (it) |
| DE (1) | DE60330136D1 (it) |
| DK (1) | DK1501931T3 (it) |
| ES (1) | ES2336909T3 (it) |
| IT (1) | ITRM20020253A1 (it) |
| PT (1) | PT1501931E (it) |
| SI (1) | SI1501931T1 (it) |
| WO (1) | WO2003095647A2 (it) |
Families Citing this family (61)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| ES2374534T3 (es) | 1998-03-20 | 2012-02-17 | Commonwealth Scientific And Industrial Research Organisation | Control de la expresión de genes. |
| AUPP249298A0 (en) | 1998-03-20 | 1998-04-23 | Ag-Gene Australia Limited | Synthetic genes and genetic constructs comprising same I |
| 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 |
| US20080044906A1 (en) * | 2003-09-12 | 2008-02-21 | Peter Michael Waterhouse | 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 (en) | 2006-05-19 | 2007-11-21 | Academisch Ziekenhuis Leiden | Means and method for inducing exon-skipping |
| PL2049664T3 (pl) | 2006-08-11 | 2012-02-29 | Biomarin Tech Bv | Jednoniciowe oligonukleotydy komplementarne do powtarzalnych elementów do leczenia chorób genetycznych związanych z niestabilnością powtórzeń DNA |
| WO2008113773A2 (en) * | 2007-03-16 | 2008-09-25 | 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 |
| 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 |
| EP2142672B1 (en) * | 2007-03-30 | 2012-09-05 | Rutgers, The State University of New Jersey | Compositions and methods for gene silencing |
| NZ582521A (en) | 2007-07-12 | 2011-09-30 | Prosensa Technologies Bv | A conjugate comprising the amino acid sequence LGAQSNF for targeting compounds to muscle tissue |
| AU2008273094B2 (en) | 2007-07-12 | 2013-05-09 | 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 |
| WO2009054725A2 (en) | 2007-10-26 | 2009-04-30 | Academisch Ziekenhuis Leiden | 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 (en) | 2008-05-14 | 2009-11-18 | Prosensa Technologies B.V. | Method for efficient exon (44) skipping in Duchenne Muscular Dystrophy and associated means |
| ES2634118T3 (es) | 2009-02-11 | 2017-09-26 | The University Of North Carolina At Chapel Hill | Vectores de virus modificados y métodos para fabricar y utilizar los mismos |
| ES2593836T3 (es) | 2009-04-24 | 2016-12-13 | Biomarin Technologies B.V. | Oligonucleótido que comprende una inosina para tratar la 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 |
| WO2011078672A1 (en) | 2009-12-24 | 2011-06-30 | Prosensa Technologies B.V. | Molecule for treating an inflammatory disorder |
| ES2683695T3 (es) | 2010-01-12 | 2018-09-27 | The University Of North Carolina At Chapel Hill | Repeticiones terminales invertidas restrictivas para vectores virales |
| TWI541024B (zh) | 2010-09-01 | 2016-07-11 | 日本新藥股份有限公司 | 反義核酸 |
| WO2012109570A1 (en) | 2011-02-10 | 2012-08-16 | The University Of North Carolina At Chapel Hill | Viral vectors with modified transduction profiles and methods of making and using the same |
| WO2012138846A2 (en) * | 2011-04-05 | 2012-10-11 | The Regents Of The University Of California | Method and compositions comprising small rna agonist and antagonists to modulate inflammation |
| NZ627896A (en) | 2012-01-27 | 2016-11-25 | Biomarin Technologies B V | Rna modulating oligonucleotides with improved characteristics for the treatment of duchenne and becker muscular dystrophy |
| CN111621507A (zh) | 2012-09-28 | 2020-09-04 | 北卡罗来纳-查佩尔山大学 | 靶向少突胶质细胞的aav载体 |
| EP2970966A4 (en) | 2013-03-15 | 2016-10-26 | Univ North Carolina | SYNTHETIC ADENOASSOZED VIRUS INVERTED REPEAT SEQUENCES |
| DK2968605T3 (da) | 2013-03-15 | 2022-09-26 | Univ North Carolina Chapel Hill | Fremgangsmåder og sammensætninger til dobbelte glycanbindende aav-vektorer |
| AU2015349759B2 (en) | 2014-11-21 | 2022-01-06 | The University Of North Carolina At Chapel Hill | Aav vectors targeted to the central nervous system |
| AU2016206624B2 (en) | 2015-01-14 | 2020-08-06 | The University Of North Carolina At Chapel Hill | Methods and compositions for targeted gene transfer |
| HK1248554A1 (zh) | 2015-02-20 | 2018-10-19 | Board Of Regents, The University Of Texas System | 作为疫苗和载体的减毒衣原体的方法和组合物 |
| BR112018003665A2 (pt) | 2015-09-28 | 2018-09-25 | Univ North Carolina Chapel Hill | métodos e composições para vetores virais de evasão de anticorpo |
| EP3390429B1 (en) | 2015-12-14 | 2025-06-18 | The University of North Carolina at Chapel Hill | Modified capsid proteins for enhanced delivery of parvovirus vectors |
| WO2018170310A1 (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 |
| BR112020005103A2 (pt) | 2017-09-15 | 2020-09-24 | Commonwealth Scientific And Industrial Research Organisation | molécula de rna, molécula de ácido ribonucleico (rna) quimérica, polinucleotídeo isolado e/ou exógeno, vetor, célula hospedeira, polinucleotídeo, organismo não humano, método, método para produzir um organismo não humano, extrato de uma célula hospedeira, composição, método para regular de modo decrescente o nível e/ou a atividade de uma molécula de rna-alvo em um organismo, método para controlar um organismo não humano, método para tratar uma doença em um indivíduo, molécula de rna, uso de uma molécula de rna e kit |
| CA3092107A1 (en) | 2018-02-28 | 2019-09-06 | The University Of North Carolina At Chapel Hill | Methods and compositions for antibody-evading virus vectors |
| JP7425738B2 (ja) | 2018-04-03 | 2024-01-31 | ギンコ バイオワークス インコーポレイテッド | 眼組織を標的とするウイルスベクター |
| EP3774852A1 (en) | 2018-04-03 | 2021-02-17 | Stridebio, Inc. | Antibody-evading virus vectors |
| BR112020020266A2 (pt) | 2018-04-03 | 2021-01-19 | Stridebio, Inc. | Vetores de vírus com evasão de anticorpos |
| EP3807297A4 (en) | 2018-06-12 | 2022-03-23 | The University of North Carolina at Chapel Hill | SYNTHETIC LIVER ADENO-ASSOCIATED VIRUS CAPSIDS AND USES THEREOF |
| 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 |
| AU2020241888A1 (en) | 2019-03-21 | 2021-09-30 | Ginkgo Bioworks, Inc. | Recombinant adeno-associated virus vectors |
| CA3137106A1 (en) | 2019-04-26 | 2020-10-29 | The University Of North Carolina At Chapel Hill | Methods and compositions for dual glycan binding aav2.5 vector |
| CN114787180A (zh) | 2019-10-17 | 2022-07-22 | 斯特里迪比奥公司 | 用于治疗c型尼曼-匹克病的腺相关病毒载体 |
| US12209251B2 (en) | 2020-05-05 | 2025-01-28 | The University Of North Carolina At Chapel Hill | Modified adeno-associated virus 5 capsids and uses thereof |
| IL300728A (en) | 2020-08-19 | 2023-04-01 | Sarepta Therapeutics Inc | Adeno-associated virus vectors for the treatment of Rett syndrome |
| 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 |
| 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 |
| 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 |
| AU2022208480A1 (en) | 2021-01-15 | 2023-08-17 | Outpace Bio, Inc. | Small molecule-regulated gene expression system |
| JP2024515626A (ja) | 2021-04-16 | 2024-04-10 | 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 | 血液脳関門を横断し低減された液性応答を惹起する合理的ポリプロイドaavビリオン |
| 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 |
| AU2023228712A1 (en) | 2022-03-04 | 2024-10-17 | Regeneron Pharmaceuticals, Inc. | Compositions and methods comprising engineered small nuclear rna (snrna) |
| AR128918A1 (es) * | 2022-03-30 | 2024-06-26 | Biomarin Pharm Inc | Oligonucleótidos que omiten el exón 51 de distrofina y sus aplicaciones |
| 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 |
| WO2024124019A2 (en) | 2022-12-07 | 2024-06-13 | Ginkgo Bioworks, Inc. | Aav vectors targeting hematopoietic stem cells |
| WO2025079025A1 (en) | 2023-10-13 | 2025-04-17 | Takeda Pharmaceutical Company Limited | Ides-rapamycin conjugate in aav gene therapy |
Family Cites Families (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| 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/it unknown
-
2003
- 2003-05-06 AT AT03749984T patent/ATE449170T1/de active
- 2003-05-06 EP EP03749984A patent/EP1501931B1/en not_active Expired - Lifetime
- 2003-05-06 DK DK03749984.5T patent/DK1501931T3/da active
- 2003-05-06 WO PCT/IT2003/000273 patent/WO2003095647A2/en not_active Application Discontinuation
- 2003-05-06 DE DE60330136T patent/DE60330136D1/de not_active Expired - Lifetime
- 2003-05-06 EP EP09176221.1A patent/EP2151497B1/en not_active Expired - Lifetime
- 2003-05-06 AU AU2003231928A patent/AU2003231928A1/en not_active Abandoned
- 2003-05-06 ES ES03749984T patent/ES2336909T3/es not_active Expired - Lifetime
- 2003-05-06 PT PT03749984T patent/PT1501931E/pt unknown
- 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 |
|---|---|
| WO2003095647A2 (en) | 2003-11-20 |
| DE60330136D1 (de) | 2009-12-31 |
| ITRM20020253A1 (it) | 2003-11-10 |
| ES2336909T3 (es) | 2010-04-19 |
| DK1501931T3 (da) | 2010-03-29 |
| AU2003231928A1 (en) | 2003-11-11 |
| AU2003231928A8 (en) | 2003-11-11 |
| EP2151497A2 (en) | 2010-02-10 |
| EP1501931A2 (en) | 2005-02-02 |
| EP1501931B1 (en) | 2009-11-18 |
| PT1501931E (pt) | 2010-02-23 |
| ATE449170T1 (de) | 2009-12-15 |
| SI1501931T1 (sl) | 2010-03-31 |
| EP2151497A3 (en) | 2010-03-10 |
| EP2151497B1 (en) | 2014-01-08 |
| WO2003095647A3 (en) | 2004-02-19 |
| CY1109746T1 (el) | 2014-09-10 |
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