CY1109746T1 - Χιμαιρικα μορια snrna που φερουν αντιπληροφοριακες αλληλουχιες εναντι των συνδεσμων συρραφης του γονιδιου δυστροφινης και θεραπευτικες εφαρμογες αυτων - Google Patents

Χιμαιρικα μορια snrna που φερουν αντιπληροφοριακες αλληλουχιες εναντι των συνδεσμων συρραφης του γονιδιου δυστροφινης και θεραπευτικες εφαρμογες αυτων

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Publication number
CY1109746T1
CY1109746T1 CY20101100102T CY101100102T CY1109746T1 CY 1109746 T1 CY1109746 T1 CY 1109746T1 CY 20101100102 T CY20101100102 T CY 20101100102T CY 101100102 T CY101100102 T CY 101100102T CY 1109746 T1 CY1109746 T1 CY 1109746T1
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Cyprus
Prior art keywords
gene
therapeutic apparatus
antisense sequences
dystrophin gene
sequences against
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CY20101100102T
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English (en)
Inventor
I Bozzoni
Angelis Fg De
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Universita' Degli Studi Di Roma "La Sapienza"
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Application filed by Universita' Degli Studi Di Roma "La Sapienza" filed Critical Universita' Degli Studi Di Roma "La Sapienza"
Publication of CY1109746T1 publication Critical patent/CY1109746T1/el

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    • 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

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  • 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)

Abstract

Περιγράφεται γονίδιο το οποίο κωδικεύει snRNA τροποποιημένο έτσι ώστε να περιέχει αντιπληροφοριακή αλληλουχία συμπληρωματική προς τουλάχιστον έναν των δύο συνδέσμων συρραφής στο 5' και/ή 3' ενός των εξονίων που κωδικεύουν πρωτεΐνη θεραπευτικού ενδιαφέροντος, του οποίου η αλληλουχία θα πρέπει να παρακαμφθεί κατά τη διεργασία συρραφής που μετατρέπει το προ-mRNA στο ώριμο mRNA. Ιδιαιτέρως καλά αποτελέσματα δίδει το γονίδιο το οποίο περιέχει τομείς συμπληρωματικούς και στους δύο συνδέσμους συρραφής. Εάν το γονίδιο εισαχθεί σε κατάλληλους φορείς, τότε είναι χρήσιμο στη γονιδιακή θεραπεία, ιδίως στην αγωγή μυικών δυστροφιών Duchenne, δυστροφικών παθήσεων.
CY20101100102T 2002-05-08 2010-02-03 Χιμαιρικα μορια snrna που φερουν αντιπληροφοριακες αλληλουχιες εναντι των συνδεσμων συρραφης του γονιδιου δυστροφινης και θεραπευτικες εφαρμογες αυτων CY1109746T1 (el)

Applications Claiming Priority (2)

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

Publications (1)

Publication Number Publication Date
CY1109746T1 true CY1109746T1 (el) 2014-09-10

Family

ID=11456291

Family Applications (1)

Application Number Title Priority Date Filing Date
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

Country Status (11)

Country Link
EP (2) EP1501931B1 (el)
AT (1) ATE449170T1 (el)
AU (1) AU2003231928A1 (el)
CY (1) CY1109746T1 (el)
DE (1) DE60330136D1 (el)
DK (1) DK1501931T3 (el)
ES (1) ES2336909T3 (el)
IT (1) ITRM20020253A1 (el)
PT (1) PT1501931E (el)
SI (1) SI1501931T1 (el)
WO (1) WO2003095647A2 (el)

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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
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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
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NZ584793A (en) 2007-10-26 2012-05-25 Academisch Ziekenhuis Leiden Means and methods for counteracting muscle disorders
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Also Published As

Publication number Publication date
WO2003095647A2 (en) 2003-11-20
ES2336909T3 (es) 2010-04-19
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
ITRM20020253A1 (it) 2003-11-10
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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