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
Application number
IT2002RM000253A
Other languages
English (en)
Inventor
Irene Bozzoni
Angelis Fernanda Gabriella De
Original Assignee
Univ Roma
Priority date (The priority date 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 date listed.)
Filing date
Publication date
Application filed by Univ Roma filed Critical Univ Roma
Priority to IT2002RM000253A priority Critical patent/ITRM20020253A1/it
Publication of ITRM20020253A0 publication Critical patent/ITRM20020253A0/it
Priority to PT03749984T priority patent/PT1501931E/pt
Priority to SI200331749T priority patent/SI1501931T1/sl
Priority to EP09176221.1A priority patent/EP2151497B1/en
Priority to DK03749984.5T priority patent/DK1501931T3/da
Priority to PCT/IT2003/000273 priority patent/WO2003095647A2/en
Priority to EP03749984A priority patent/EP1501931B1/en
Priority to AT03749984T priority patent/ATE449170T1/de
Priority to ES03749984T priority patent/ES2336909T3/es
Priority to AU2003231928A priority patent/AU2003231928A1/en
Priority to DE60330136T priority patent/DE60330136D1/de
Publication of ITRM20020253A1 publication Critical patent/ITRM20020253A1/it
Priority to CY20101100102T priority patent/CY1109746T1/el

Links

Classifications

    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K2319/00Fusion polypeptide
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/11Antisense
    • C12N2310/111Antisense 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)
IT2002RM000253A 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. ITRM20020253A1 (it)

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)

* Cited by examiner, † Cited by third party
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)

* Cited by examiner, † Cited by third party
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

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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