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
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 EP09176221.1A priority patent/EP2151497B1/en
Priority to AT03749984T priority patent/ATE449170T1/de
Priority to DK03749984.5T priority patent/DK1501931T3/da
Priority to PT03749984T priority patent/PT1501931E/pt
Priority to SI200331749T priority patent/SI1501931T1/sl
Priority to DE60330136T priority patent/DE60330136D1/de
Priority to ES03749984T priority patent/ES2336909T3/es
Priority to PCT/IT2003/000273 priority patent/WO2003095647A2/en
Priority to AU2003231928A priority patent/AU2003231928A1/en
Priority to EP03749984A priority patent/EP1501931B1/en
Publication of ITRM20020253A1 publication Critical patent/ITRM20020253A1/it
Priority to CY20101100102T priority patent/CY1109746T1/el

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)
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.
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
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
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
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
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
SI200331749T SI1501931T1 (sl) 2002-05-08 2003-05-06 Himerne molekule snrna ki nosijo protismiselne sekvence proti mestu cepitve distrofinskega gena innjihove terapevtske aplikacije
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
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.
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
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 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) 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)

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CN1796556A (zh) 1998-03-20 2006-07-05 联邦科学和工业研究组织 控制基因表达
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 (en) 2006-05-19 2007-11-21 Academisch Ziekenhuis Leiden Means and method for inducing exon-skipping
WO2008018795A1 (en) 2006-08-11 2008-02-14 Prosensa Technologies 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
US8907075B2 (en) 2007-03-30 2014-12-09 Samuel Ian Gunderson Compositions and methods for gene silencing
WO2008121963A2 (en) * 2007-03-30 2008-10-09 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
US9441221B2 (en) 2007-03-30 2016-09-13 Rutgers, The State University Of New Jersey Compositions and methods for gene silencing
DK2167136T3 (en) 2007-07-12 2016-07-25 Biomarin Tech Bv Molecules for targeting compounds at different selected organs or tissues
EP2167135A2 (en) 2007-07-12 2010-03-31 Prosensa Technologies B.V. Molecules for targeting compounds to various selected organs, tissues or tumor cells
NZ584793A (en) 2007-10-26 2012-05-25 Academisch Ziekenhuis Leiden Means and methods for counteracting muscle disorders
USRE48468E1 (en) 2007-10-26 2021-03-16 Biomarin Technologies B.V. 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
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
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
AU2010335039B2 (en) 2009-12-24 2015-03-26 Academisch Ziekenhuis Leiden H.O.D.N. Lumc 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
US9409953B2 (en) 2011-02-10 2016-08-09 The University Of North Carolina At Chapel Hill Viral vectors with modified transduction profiles and methods of making and using the same
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
EP2806900B1 (en) 2012-01-27 2021-12-15 BioMarin Technologies B.V. Rna modulating oligonucleotides with improved characteristics for the treatment of duchenne and becker muscular dystrophy
CA2885544A1 (en) 2012-09-28 2014-04-03 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
EP3517612A1 (en) 2013-03-15 2019-07-31 The University of North Carolina At Chapel Hill Synthetic adeno-associated virus inverted terminal repeats
EP4344741A2 (en) 2014-11-21 2024-04-03 The University of North Carolina at Chapel Hill Aav vectors targeted to the central nervous system
CA2972807C (en) 2015-01-14 2024-01-16 The University Of North Carolina At Chapel Hill Methods and compositions for targeted gene transfer
US10596247B2 (en) 2015-02-20 2020-03-24 Board Of Regents, The University Of Texas System Methods and compositions for attenuated chlamydia as vaccine and vector
IL292830B2 (en) 2015-09-28 2023-06-01 Univ Florida Methods and compositions for stealth virus antibody vectors
AU2018234695A1 (en) 2017-03-15 2019-09-12 The University Of North Carolina At Chapel Hill Polyploid adeno-associated virus vectors and methods of making and using the same
AU2019247746A1 (en) 2018-04-03 2020-10-15 Ginkgo Bioworks, Inc. Antibody-evading virus vectors
CN112533644A (zh) 2018-04-03 2021-03-19 斯特里迪比奥公司 靶向眼组织的病毒载体
CA3094311A1 (en) 2018-04-03 2019-10-10 Stridebio, Inc. Antibody-evading virus vectors
CA3104113A1 (en) 2018-06-22 2019-12-26 Asklepios Biopharmaceutical, Inc. Vectors for gene delivery that persist within cells
CN113727992A (zh) 2019-03-21 2021-11-30 斯特里迪比奥公司 重组腺相关病毒载体
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
CA3157700A1 (en) 2019-10-17 2021-04-22 Stridebio, Inc. Adeno-associated viral vectors for treatment of niemann-pick disease type c
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
CN116490514A (zh) 2020-10-28 2023-07-25 北卡罗来纳-查佩尔山大学 用于双重聚糖结合aav2.5载体的方法和组合物
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
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Also Published As

Publication number Publication date
WO2003095647A2 (en) 2003-11-20
ES2336909T3 (es) 2010-04-19
CY1109746T1 (el) 2014-09-10
AU2003231928A1 (en) 2003-11-11
EP1501931A2 (en) 2005-02-02
WO2003095647A3 (en) 2004-02-19
SI1501931T1 (sl) 2010-03-31
ATE449170T1 (de) 2009-12-15
AU2003231928A8 (en) 2003-11-11
DE60330136D1 (de) 2009-12-31
EP2151497A2 (en) 2010-02-10
ITRM20020253A0 (it) 2002-05-08
PT1501931E (pt) 2010-02-23
EP1501931B1 (en) 2009-11-18
EP2151497B1 (en) 2014-01-08
DK1501931T3 (da) 2010-03-29
EP2151497A3 (en) 2010-03-10

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