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. |
| SI200331749T SI1501931T1 (sl) | 2002-05-08 | 2003-05-06 | Himerne molekule snrna ki nosijo protismiselne sekvence proti mestu cepitve distrofinskega gena innjihove terapevtske aplikacije |
| 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 |
| 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 |
| 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 |
| 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 |
| 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 |
| 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 |
| 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. |
| 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 (61)
| 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 |
| 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 |
| 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 |
| 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 |
| US8343941B2 (en) | 2007-03-30 | 2013-01-01 | 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 |
| ES2564563T3 (es) | 2007-10-26 | 2016-03-23 | Academisch Ziekenhuis Leiden | Medios y métodos para contrarrestar trastornos del músculo |
| USRE48468E1 (en) | 2007-10-26 | 2021-03-16 | Biomarin Technologies B.V. | Means and methods for counteracting muscle disorders |
| AU2009210872A1 (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 |
| WO2010093784A2 (en) | 2009-02-11 | 2010-08-19 | The University Of North Carolina At Chapel Hill | Modified virus vectors and methods of making and using the same |
| JP2012524540A (ja) | 2009-04-24 | 2012-10-18 | プロセンサ テクノロジーズ ビー．ブイ． | Ｄｍｄを処置するためのイノシンを含むオリゴヌクレオチド |
| 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 |
| EP2524037B1 (en) | 2010-01-12 | 2018-05-16 | The University Of North Carolina At Chapel Hill | Restrictive inverted terminal repeats for viral vectors |
| 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 |
| BR112014018427B1 (pt) | 2012-01-27 | 2021-11-03 | Biomarin Technologies B.V. | Oligonucleotídeos moduladores de rna com características melhoradas para o tratamento da distrofia muscular de duchenne e becker |
| EP2900686B1 (en) | 2012-09-28 | 2020-06-10 | The University of North Carolina At Chapel Hill | Aav vectors targeted to oligodendrocytes |
| PL2968605T3 (pl) | 2013-03-15 | 2022-11-07 | The University Of North Carolina At Chapel Hill | Metody i kompozycje dla wektorów aav podwójnie wiążących glikan |
| WO2014143932A1 (en) | 2013-03-15 | 2014-09-18 | The University Of North Carolina At Chapel Hill | Synthetic adeno-associated virus inverted terminal repeats |
| JP2017536116A (ja) | 2014-11-21 | 2017-12-07 | ザ ユニバーシティ オブ ノース カロライナ アット チャペル ヒル | 中枢神経系を標的化したａａｖベクター |
| CA2972807C (en) | 2015-01-14 | 2024-01-16 | 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 |
| PT3356390T (pt) | 2015-09-28 | 2021-04-21 | Univ Florida | Métodos e composições para vetores virais que se evadem a anticorpos |
| CN108602859B (zh) | 2015-12-14 | 2023-05-26 | 北卡罗来纳大学教堂山分校 | 增强细小病毒载体递送的修饰衣壳蛋白 |
| JP7132934B2 (ja) | 2017-03-15 | 2022-09-07 | ザ ユニバーシティ オブ ノース カロライナ アット チャペル ヒル | ポリプロイド性アデノ随伴ウイルスベクターならびにその作製および使用の方法 |
| IL273245B2 (en) | 2017-09-15 | 2025-03-01 | Commw Scient Ind Res Org | RNA molecules |
| AU2019227896A1 (en) | 2018-02-28 | 2020-08-20 | The University Of North Carolina At Chapel Hill | Methods and compositions for antibody-evading virus vectors |
| EP3773743A1 (en) | 2018-04-03 | 2021-02-17 | Stridebio, Inc. | Virus vectors for targeting ophthalmic tissues |
| WO2019195449A1 (en) | 2018-04-03 | 2019-10-10 | Stridebio, Inc. | Antibody-evading virus vectors |
| MX2020010466A (es) | 2018-04-03 | 2021-01-08 | Vectores de virus que evitan anticuerpos. | |
| CA3103186A1 (en) | 2018-06-12 | 2019-12-19 | The University Of North Carolina At Chapel Hill | Synthetic liver-tropic adeno-associated virus capsids and uses thereof |
| US20210269828A1 (en) | 2018-06-22 | 2021-09-02 | Asklepios Biopharmaceutical, Inc. | Vectors for gene delivery that persist within cells |
| MX2021011468A (es) | 2019-03-21 | 2021-12-15 | Vectores de virus adenoasociados recombinantes. | |
| 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 |
| WO2021076925A1 (en) | 2019-10-17 | 2021-04-22 | Stridebio, Inc. | Adeno-associated viral vectors for treatment of niemann-pick disease type c |
| MX2022013819A (es) | 2020-05-05 | 2023-01-30 | Univ North Carolina Chapel Hill | Capsides de virus adeno-asociado 5 modificado y usos del mismo. |
| US12104163B2 (en) | 2020-08-19 | 2024-10-01 | Sarepta Therapeutics, Inc. | Adeno-associated virus vectors for 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 |
| JP2023547992A (ja) | 2020-10-28 | 2023-11-15 | 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 |
| 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 |
| CA3216491A1 (en) | 2021-04-16 | 2022-10-20 | Asklepios Biopharmaceutical, Inc. | Rational polyploid aav virions that cross the blood brain barrier and elicit reduced humoral response |
| WO2023150687A1 (en) | 2022-02-04 | 2023-08-10 | Ginkgo Bioworks, Inc. | Recombinant adeno-associated virus vectors, and methods of use thereof |
| EP4486888A1 (en) | 2022-03-04 | 2025-01-08 | Locanabio, Inc. | Compositions and methods comprising engineered small nuclear rna (snrna) |
| TW202342070A (zh) * | 2022-03-30 | 2023-11-01 | 美商拜奧馬林製藥公司 | 肌萎縮蛋白外顯子跳躍寡核苷酸 |
| 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 WO PCT/IT2003/000273 patent/WO2003095647A2/en not_active Application Discontinuation
- 2003-05-06 ES ES03749984T patent/ES2336909T3/es not_active Expired - Lifetime
- 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 DE DE60330136T patent/DE60330136D1/de not_active Expired - Lifetime
- 2003-05-06 SI SI200331749T patent/SI1501931T1/sl unknown
- 2003-05-06 EP EP03749984A patent/EP1501931B1/en not_active Expired - Lifetime
- 2003-05-06 AU AU2003231928A patent/AU2003231928A1/en not_active Abandoned
- 2003-05-06 DK DK03749984.5T patent/DK1501931T3/da active
-
2010
- 2010-02-03 CY CY20101100102T patent/CY1109746T1/el unknown
Also Published As
| Publication number | Publication date |
|---|---|
| EP2151497A3 (en) | 2010-03-10 |
| EP1501931B1 (en) | 2009-11-18 |
| ITRM20020253A0 (it) | 2002-05-08 |
| WO2003095647A2 (en) | 2003-11-20 |
| ES2336909T3 (es) | 2010-04-19 |
| CY1109746T1 (el) | 2014-09-10 |
| WO2003095647A3 (en) | 2004-02-19 |
| DK1501931T3 (da) | 2010-03-29 |
| ATE449170T1 (de) | 2009-12-15 |
| DE60330136D1 (de) | 2009-12-31 |
| AU2003231928A1 (en) | 2003-11-11 |
| SI1501931T1 (sl) | 2010-03-31 |
| PT1501931E (pt) | 2010-02-23 |
| EP1501931A2 (en) | 2005-02-02 |
| EP2151497B1 (en) | 2014-01-08 |
| AU2003231928A8 (en) | 2003-11-11 |
| EP2151497A2 (en) | 2010-02-10 |
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