EP2152873A2 - Technique de regulation de l'expression genetique et arn non codant a visee diagnostique et therapeutique - Google Patents

Technique de regulation de l'expression genetique et arn non codant a visee diagnostique et therapeutique

Info

Publication number
EP2152873A2
EP2152873A2 EP08735425A EP08735425A EP2152873A2 EP 2152873 A2 EP2152873 A2 EP 2152873A2 EP 08735425 A EP08735425 A EP 08735425A EP 08735425 A EP08735425 A EP 08735425A EP 2152873 A2 EP2152873 A2 EP 2152873A2
Authority
EP
European Patent Office
Prior art keywords
seq
sequence
nucleic acid
acid molecule
promoter
Prior art date
Legal status (The legal status 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 status listed.)
Withdrawn
Application number
EP08735425A
Other languages
German (de)
English (en)
Inventor
Aldo Pagano
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
BIORIGEN Srl
Original Assignee
BIORIGEN Srl
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 BIORIGEN Srl filed Critical BIORIGEN Srl
Publication of EP2152873A2 publication Critical patent/EP2152873A2/fr
Withdrawn legal-status Critical Current

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
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/8509Vectors or expression systems specially adapted for eukaryotic hosts for animal cells for producing genetically modified animals, e.g. transgenic
    • 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
    • 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
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • AHUMAN NECESSITIES
    • A01AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01KANIMAL HUSBANDRY; CARE OF BIRDS, FISHES, INSECTS; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2267/00Animals characterised by purpose
    • A01K2267/03Animal model, e.g. for test or diseases
    • A01K2267/0306Animal model for genetic diseases
    • A01K2267/0312Animal model for Alzheimer's disease
    • 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
    • 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
    • C12N2310/30Chemical structure
    • C12N2310/35Nature of the modification
    • C12N2310/351Conjugate
    • C12N2310/3519Fusion with another nucleic acid
    • 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
    • C12N2310/50Physical structure
    • C12N2310/53Physical structure partially self-complementary or closed
    • 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
    • C12N2330/00Production
    • C12N2330/10Production naturally occurring

Landscapes

  • Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Wood Science & Technology (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Physics & Mathematics (AREA)
  • Veterinary Medicine (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
  • Peptides Or Proteins (AREA)

Abstract

Cette invention concerne une molécule d'acide nucléique comprenant séquentiellement : une séquence dérivée d'un petit ARN 7SL comprenant au moins le domaine de fixation aux protéines srp9 et srp14 du ribonucléocomplexe 7SL ; une séquence identique ou complémentaire d'une séquence cible ; un promoteur pol III de type III. L'invention concerne également les dérivés de ladite molécule en vue d'une utilisation médicale et diagnostique.
EP08735425A 2007-03-16 2008-03-14 Technique de regulation de l'expression genetique et arn non codant a visee diagnostique et therapeutique Withdrawn EP2152873A2 (fr)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US89542607P 2007-03-16 2007-03-16
PCT/EP2008/053132 WO2008113773A2 (fr) 2007-03-16 2008-03-14 Technique de régulation de l'expression génétique et arn non codant à visée diagnostique et thérapeutique

Publications (1)

Publication Number Publication Date
EP2152873A2 true EP2152873A2 (fr) 2010-02-17

Family

ID=39691031

Family Applications (1)

Application Number Title Priority Date Filing Date
EP08735425A Withdrawn EP2152873A2 (fr) 2007-03-16 2008-03-14 Technique de regulation de l'expression genetique et arn non codant a visee diagnostique et therapeutique

Country Status (2)

Country Link
EP (1) EP2152873A2 (fr)
WO (1) WO2008113773A2 (fr)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
ES2855577T3 (es) * 2011-03-30 2021-09-23 Transine Therapeutics Ltd Molécula de ácido nucleico funcional y uso de la misma
WO2014149882A1 (fr) * 2013-03-15 2014-09-25 University Of Iowa Research Foundation Compositions de promoteur

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2007034527A2 (fr) * 2005-09-19 2007-03-29 Universita Degli Studi Di Genova Unites transcriptionnelles du type gene arnsn et utilisations

Family Cites Families (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
IT1283876B1 (it) * 1996-01-12 1998-05-07 Univ Roma Molecole chimeriche ribozima-snrna ad attivita' catalitica per rna a localizzazione nucleare
US5814500A (en) * 1996-10-31 1998-09-29 The Johns Hopkins University School Of Medicine Delivery construct for antisense nucleic acids and methods of use
US6692910B2 (en) * 1997-07-28 2004-02-17 The University Of Connecticut Inhibition of a target messenger RNA with a modified U1 small nuclear RNA
CA2376284A1 (fr) * 1999-06-21 2000-12-28 Murdoch Childrens Research Institute Methode de prophylaxie et/ou de traitement de troubles cliniques
US20020058287A1 (en) * 2000-03-10 2002-05-16 Whitehead Institute For Biomedical Research Novel small nuclear RNA vectors and uses therefor
ITRM20020253A1 (it) * 2002-05-08 2003-11-10 Univ Roma Molecole chimeriche di snrna con sequenze antisenso per le giunzioni di splicing del gene della distrofina e applicazioni terapeutiche.
WO2004005458A2 (fr) * 2002-06-13 2004-01-15 Regulome Corporation Sites fonctionnels
US20050272043A1 (en) * 2003-07-24 2005-12-08 Roth Richard B Methods for identifying risk of breast cancer and treatments thereof
WO2005026356A1 (fr) * 2003-09-12 2005-03-24 Commonwealth Scientific And Industrial Research Organisation Molecules modifiees d'acide nucleique represseur de gene et leurs utilisation
WO2006022639A1 (fr) * 2004-07-21 2006-03-02 Applera Corporation Polymorphismes génétiques associés à la maladie d’alzheimer, procédés de détection et leurs utilisations
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

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2007034527A2 (fr) * 2005-09-19 2007-03-29 Universita Degli Studi Di Genova Unites transcriptionnelles du type gene arnsn et utilisations

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
LIU SONGKAI ET AL: "Inhibition of HIV-1 multiplication by antisense U7 snRNAs and siRNAs targeting cyclophilin A", NUCLEIC ACIDS RESEARCH, OXFORD UNIVERSITY PRESS, SURREY, GB, vol. 32, no. 12, 14 July 2004 (2004-07-14), pages 3752 - 3759, XP002376976, ISSN: 0305-1048 *

Also Published As

Publication number Publication date
WO2008113773A2 (fr) 2008-09-25
WO2008113773A3 (fr) 2008-12-24

Similar Documents

Publication Publication Date Title
Alexander et al. MicroRNA-199a is induced in dystrophic muscle and affects WNT signaling, cell proliferation, and myogenic differentiation
Tanackovic et al. Human splicing factor SF3a, but not SF1, is essential for pre-mRNA splicing in vivo
Skourti-Stathaki et al. Human senataxin resolves RNA/DNA hybrids formed at transcriptional pause sites to promote Xrn2-dependent termination
Fragiadaki et al. High doses of TGF-β potently suppress type I collagen via the transcription factor CUX1
Tian et al. MiR-320 regulates cardiomyocyte apoptosis induced by ischemia–reperfusion injury by targeting AKIP1
US9353370B2 (en) Functional nucleic acid molecule and use thereof
Peacey et al. Targeting a pre-mRNA structure with bipartite antisense molecules modulates tau alternative splicing
Roshan et al. Regulation of BACE1 by miR-29a/b in a cellular model of Spinocerebellar Ataxia 17
BR112020005287A2 (pt) composições e métodos para edição de gene ttr e tratar amiloidose attr
US11814643B2 (en) Enhanced sleeping beauty transposons, kits and methods of transposition
Sofuku et al. Transcription profiling demonstrates epigenetic control of non-retroviral RNA virus-derived elements in the human genome
WO2012049665A1 (fr) Molécule u1snrna d'origine humaine modifiée, gène codant la molécule u1snrna d'origine humaine modifiée, vecteur d'expression contenant le gène et utilisation en thérapie génique
US20210254163A1 (en) Exonic splicing enhancers and exonic splicing silencers
D'Souza et al. Arginine/serine-rich protein interaction domain-dependent modulation of a tau exon 10 splicing enhancer: altered interactions and mechanisms for functionally antagonistic FTDP-17 mutations Δ280K and N279K
Größl et al. A novel artificial microRNA expressing AAV vector for phospholamban silencing in cardiomyocytes improves Ca2+ uptake into the sarcoplasmic reticulum
ES2659845A1 (es) Modulación de microRNAs contra la distrofia miotónica tipo 1 y antagonistas de microRNAs para ello
Mancuso et al. A cryptic promoter in the first exon of the SPG4 gene directs the synthesis of the 60-kDa spastin isoform
Urbanelli et al. Characterization of human Enah gene
US20230323391A1 (en) Transgene expression system
Yu et al. Transcriptional regulation of human FE65, a ligand of Alzheimer's disease amyloid precursor protein, by Sp1
Gall-Duncan et al. Antagonistic roles of canonical and Alternative-RPA in disease-associated tandem CAG repeat instability
EP2152873A2 (fr) Technique de regulation de l'expression genetique et arn non codant a visee diagnostique et therapeutique
Xu et al. A group of tissue‐specific microRNAs contribute to the silencing of CUX1 in different cell lineages during development
Winkelsas et al. Targeting the 5′ untranslated region of SMN2 as a therapeutic strategy for spinal muscular atrophy
WO2013017542A1 (fr) Procédé pour la prévention ou le traitement d'un affaiblissement de la mémoire et composition pharmaceutique utile à cet effet

Legal Events

Date Code Title Description
PUAI Public reference made under article 153(3) epc to a published international application that has entered the european phase

Free format text: ORIGINAL CODE: 0009012

17P Request for examination filed

Effective date: 20091015

AK Designated contracting states

Kind code of ref document: A2

Designated state(s): AT BE BG CH CY CZ DE DK EE ES FI FR GB GR HR HU IE IS IT LI LT LU LV MC MT NL NO PL PT RO SE SI SK TR

AX Request for extension of the european patent

Extension state: AL BA MK RS

17Q First examination report despatched

Effective date: 20100330

GRAP Despatch of communication of intention to grant a patent

Free format text: ORIGINAL CODE: EPIDOSNIGR1

INTG Intention to grant announced

Effective date: 20140710

STAA Information on the status of an ep patent application or granted ep patent

Free format text: STATUS: THE APPLICATION IS DEEMED TO BE WITHDRAWN

18D Application deemed to be withdrawn

Effective date: 20141001