ITRM20020253A1 - 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
- ITRM20020253A1 ITRM20020253A1 IT2002RM000253A ITRM20020253A ITRM20020253A1 IT RM20020253 A1 ITRM20020253 A1 IT RM20020253A1 IT 2002RM000253 A IT2002RM000253 A IT 2002RM000253A IT RM20020253 A ITRM20020253 A IT RM20020253A IT RM20020253 A1 ITRM20020253 A1 IT RM20020253A1
- Authority
- IT
- Italy
- Prior art keywords
- gene
- snrna
- dystrophine
- therapeutic applications
- antisense sequences
- Prior art date
Links
- 108090000623 proteins and genes Proteins 0.000 title abstract 5
- 230000000692 anti-sense effect Effects 0.000 title abstract 2
- 230000001225 therapeutic effect Effects 0.000 title abstract 2
- 108091029842 small nuclear ribonucleic acid Proteins 0.000 title 1
- 230000000295 complement effect Effects 0.000 abstract 2
- 206010013801 Duchenne Muscular Dystrophy Diseases 0.000 abstract 1
- 108700024394 Exon Proteins 0.000 abstract 1
- 108091027974 Mature messenger RNA Proteins 0.000 abstract 1
- 102000039471 Small Nuclear RNA Human genes 0.000 abstract 1
- 108020004688 Small Nuclear RNA Proteins 0.000 abstract 1
- 201000010099 disease Diseases 0.000 abstract 1
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 abstract 1
- 238000001415 gene therapy Methods 0.000 abstract 1
- 108020004999 messenger RNA Proteins 0.000 abstract 1
- 238000000034 method Methods 0.000 abstract 1
- 102000004169 proteins and genes Human genes 0.000 abstract 1
- 239000013598 vector Substances 0.000 abstract 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. |
| 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. |
| 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 |
| 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 |
| 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 |
| 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 |
| 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 |
| 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 |
| SI200331749T SI1501931T1 (sl) | 2002-05-08 | 2003-05-06 | Himerne molekule snrna ki nosijo protismiselne sekvence proti mestu cepitve distrofinskega gena innjihove terapevtske aplikacije |
| 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 |
| 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 |
| 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 ITRM20020253A0 (it) | 2002-05-08 |
| ITRM20020253A1 true 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) | EP2151497B1 (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 (63)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO1999049029A1 (en) | 1998-03-20 | 1999-09-30 | Benitec Australia Ltd | Control of gene expression |
| 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 |
| WO2005026356A1 (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 (en) | 2006-05-19 | 2007-11-21 | Academisch Ziekenhuis Leiden | Means and method for inducing exon-skipping |
| AU2007282224B2 (en) | 2006-08-11 | 2013-08-29 | Vico Therapeutics B.V. | Methods and means for treating DNA repeat instability associated genetic disorders |
| EP2152873A2 (en) * | 2007-03-16 | 2010-02-17 | Biorigen S.r.l | Gene expression regulation technology and noncoding rnas for diagnosis and therapy |
| US8343941B2 (en) | 2007-03-30 | 2013-01-01 | 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 |
| EP2142672B1 (en) * | 2007-03-30 | 2012-09-05 | 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 |
| CA2693742A1 (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 |
| AU2008317566B2 (en) | 2007-10-26 | 2014-05-01 | Academisch Ziekenhuis Leiden | 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 |
| 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 |
| EP2396343B1 (en) | 2009-02-11 | 2017-05-17 | The University of North Carolina At Chapel Hill | Modified virus vectors and methods of making and using the same |
| CA2759899A1 (en) | 2009-04-24 | 2010-10-28 | 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 |
| 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 |
| CN112251436A (zh) | 2012-01-27 | 2021-01-22 | 比奥马林技术公司 | 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 |
| US9636370B2 (en) | 2012-09-28 | 2017-05-02 | The University Of North Carolina At Chapel Hill | AAV vectors targeted to oligodendrocytes |
| US9447433B2 (en) | 2013-03-15 | 2016-09-20 | The University Of North Carolina At Chapel Hill | Synthetic adeno-associated virus inverted terminal repeats |
| JP6396988B2 (ja) | 2013-03-15 | 2018-09-26 | 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 | 二重グリカン結合性aavベクターのための方法および組成物 |
| WO2016081811A1 (en) | 2014-11-21 | 2016-05-26 | 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 |
| MX2017010698A (es) | 2015-02-20 | 2018-04-30 | Univ Texas | Metodos y composiciones para chlamydia atenuada como vacuna y vector. |
| BR112018003665A2 (en) | 2015-09-28 | 2018-09-25 | The University Of North Carolina At Chapel Hill | methods and compositions for antibody evasion viral vectors |
| EP4659816A3 (en) | 2015-12-14 | 2025-12-31 | The University of North Carolina at Chapel Hill | MODIFIED CAPSID PROTEINS FOR IMPROVED PARVOVIRUS VECTOR DELIVERY |
| CN110770346B (zh) | 2017-03-15 | 2024-01-12 | 北卡罗来纳-查佩尔山大学 | 多倍体腺相关病毒载体及其制备和使用方法 |
| IL273245B2 (en) | 2017-09-15 | 2025-03-01 | Commw Scient Ind Res Org | RNA molecules |
| BR112020016699A2 (pt) | 2018-02-28 | 2020-12-15 | The University Of North Carolina At Chapel Hill | Métodos e composições para vetores de vírus de evasão de anticorpos |
| CA3094465A1 (en) | 2018-04-03 | 2019-10-10 | Stridebio, Inc. | Virus vectors for targeting ophthalmic tissues |
| CN112543766A (zh) | 2018-04-03 | 2021-03-23 | 斯特里迪比奥公司 | 抗体逃避性病毒载体 |
| US20210363191A1 (en) | 2018-04-03 | 2021-11-25 | Stridebio, Inc. | Antibody-evading virus vectors |
| CN112566923B (zh) | 2018-06-12 | 2024-10-29 | 北卡罗来纳大学教堂山分校 | 合成嗜肝性腺相关病毒衣壳及其用途 |
| EP3810782A2 (en) | 2018-06-22 | 2021-04-28 | Asklepios Biopharmaceutical, Inc. | Vectors for gene delivery that persist within cells |
| BR112021018776A2 (pt) | 2019-03-21 | 2021-11-30 | Stridebio Inc | Vetores de vírus adenoassociado recombinante |
| EP3959227A1 (en) | 2019-04-26 | 2022-03-02 | The University of North Carolina at Chapel Hill | Methods and compositions for dual glycan binding aav2.5 vector |
| KR20220083714A (ko) | 2019-10-17 | 2022-06-20 | 스트라이드바이오 인코포레이티드 | 니만-피크 질환 c형의 치료를 위한 아데노-연관 바이러스 벡터 |
| EP4100523A4 (en) | 2020-02-04 | 2024-03-06 | Duke University | COMPOSITIONS AND METHODS FOR CIRCULAR RNA EXPRESSION |
| 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 |
| EP4200408A1 (en) | 2020-08-19 | 2023-06-28 | Sarepta Therapeutics, Inc. | Adeno-associated virus vectors for treatment of rett syndrome |
| ES3001131T3 (es) | 2020-10-28 | 2025-03-04 | Univ North Carolina Chapel Hill | Métodos y composiciones para la unión dual de glicanos al vector de AAV2.5 |
| 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 |
| US20240398993A1 (en) | 2021-01-15 | 2024-12-05 | Glenna FOIGHT | 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 |
| WO2023150687A1 (en) | 2022-02-04 | 2023-08-10 | Ginkgo Bioworks, Inc. | Recombinant adeno-associated virus vectors, and methods of use thereof |
| JP2025507958A (ja) | 2022-03-04 | 2025-03-21 | ロックアネイビオ, インコーポレイテッド | 操作された核内低分子RNA(snRNA)を含む組成物および方法 |
| AR128918A1 (es) * | 2022-03-30 | 2024-06-26 | Biomarin Pharm Inc | Oligonucleótidos que omiten el exón 51 de distrofina y sus aplicaciones |
| CN120129747A (zh) * | 2022-10-11 | 2025-06-10 | 广州瑞风生物科技有限公司 | snRNA核酸分子及其应用 |
| 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 PT PT03749984T patent/PT1501931E/pt unknown
- 2003-05-06 SI SI200331749T patent/SI1501931T1/sl unknown
- 2003-05-06 EP EP09176221.1A patent/EP2151497B1/en not_active Expired - Lifetime
- 2003-05-06 AT AT03749984T patent/ATE449170T1/de active
- 2003-05-06 DK DK03749984.5T patent/DK1501931T3/da active
- 2003-05-06 DE DE60330136T patent/DE60330136D1/de 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 WO PCT/IT2003/000273 patent/WO2003095647A2/en not_active Ceased
- 2003-05-06 EP EP03749984A patent/EP1501931B1/en not_active Expired - Lifetime
-
2010
- 2010-02-03 CY CY20101100102T patent/CY1109746T1/el unknown
Also Published As
| Publication number | Publication date |
|---|---|
| ATE449170T1 (de) | 2009-12-15 |
| PT1501931E (pt) | 2010-02-23 |
| SI1501931T1 (sl) | 2010-03-31 |
| EP2151497B1 (en) | 2014-01-08 |
| AU2003231928A8 (en) | 2003-11-11 |
| EP2151497A2 (en) | 2010-02-10 |
| CY1109746T1 (el) | 2014-09-10 |
| WO2003095647A2 (en) | 2003-11-20 |
| EP1501931B1 (en) | 2009-11-18 |
| WO2003095647A3 (en) | 2004-02-19 |
| DK1501931T3 (da) | 2010-03-29 |
| DE60330136D1 (de) | 2009-12-31 |
| ES2336909T3 (es) | 2010-04-19 |
| EP1501931A2 (en) | 2005-02-02 |
| AU2003231928A1 (en) | 2003-11-11 |
| ITRM20020253A0 (it) | 2002-05-08 |
| EP2151497A3 (en) | 2010-03-10 |
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