CY1117522T1 - Επαγωγη παρακαμψης εξωνιου σε ευκαριωτικα κυτταρα - Google Patents

Επαγωγη παρακαμψης εξωνιου σε ευκαριωτικα κυτταρα

Info

Publication number
CY1117522T1
CY1117522T1 CY20161100215T CY161100215T CY1117522T1 CY 1117522 T1 CY1117522 T1 CY 1117522T1 CY 20161100215 T CY20161100215 T CY 20161100215T CY 161100215 T CY161100215 T CY 161100215T CY 1117522 T1 CY1117522 T1 CY 1117522T1
Authority
CY
Cyprus
Prior art keywords
exon
cell
skipping
production
instance
Prior art date
Application number
CY20161100215T
Other languages
English (en)
Inventor
Ommen Garrit-Jan Boudewijn Van
Deutekom Judith Christina Theodora Van
Dunnen Johannes Theodorus Den
Original Assignee
Academisch Ziekenhuis Leiden
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
Family has litigation
First worldwide family litigation filed litigation Critical https://patents.darts-ip.com/?family=8172043&utm_source=google_patent&utm_medium=platform_link&utm_campaign=public_patent_search&patent=CY1117522(T1) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Application filed by Academisch Ziekenhuis Leiden filed Critical Academisch Ziekenhuis Leiden
Publication of CY1117522T1 publication Critical patent/CY1117522T1/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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • 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
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/10—Dispersions; Emulsions
    • A61K9/127—Liposomes
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P19/00—Drugs for skeletal disorders
    • A61P19/04—Drugs for skeletal disorders for non-specific disorders of the connective tissue
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A61P21/04—Drugs for disorders of the muscular or neuromuscular system for myasthenia gravis
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12—Antivirals
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • A61P35/04—Antineoplastic agents specific for metastasis
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P5/00—Drugs for disorders of the endocrine system
    • A61P5/14—Drugs for disorders of the endocrine system of the thyroid hormones, e.g. T3, T4
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • A61P7/04—Antihaemorrhagics; Procoagulants; Haemostatic agents; Antifibrinolytic agents
    • 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
    • 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
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; CARE OF BIRDS, FISHES, INSECTS; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2267/00—Animals characterised by purpose
    • A01K2267/03—Animal model, e.g. for test or diseases
    • 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
    • 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
    • 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
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
    • 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
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
    • 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
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
    • 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
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/33—Alteration of splicing
    • 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
    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14142—Use of virus, viral particle or viral elements as a vector virus or viral particle as vehicle, e.g. encapsulating small organic molecule

Abstract

Η παρούσα εφεύρεση παρέχει μία μέθοδο για μείωση τουλάχιστον εν μέρει της παραγωγής μιας παρεκλίνoυσας πρωτεΐνης σε ένα κύτταρο και αυτό το κύτταρο περιέχει mRNA το οποίον περιλαμβάνει εξώνια που κωδικεύουν αυτή την πρωτεΐνη, με επαγωγή της λεγομένης παράκαμψης εξωνίου σε αυτό το κύτταρο. Η παράκαμψη εξωνίου έχει σαν αποτέλεσμα ώριμο mRNA που δεν περιέχει το παρακαμφθέν εξώνιο το οποίον οδηγεί σε ένα αλλαγμένο προϊόν εάν αυτό το εξώνιο κωδικεύει αμινοξέα. Η παράκαμψη εξωνίου διεξάγεται με εφοδιασμό ενός κυττάρου με έναν παράγοντα ικανό για ειδική αναστολή σήματος έγκλεισης εξωνίου, για παράδειγμα μία αλληλουχία αναγνώρισης εξωνίου αυτού του εξωνίου. Σε αυτό το σήμα έγκλεισης εξωνίου μπορεί να παρέμβει ένα νουκλεϊνικό οξύ που περιλαμβάνει συμπληρωματικότητα για ένα μέρος αυτού του εξωνίου. Αυτό το νουκλεϊνικό οξύ το οποίον επίσης παρέχεται στο παρόν έγγραφο, μπορεί να χρησιμοποιηθεί για την παρασκευή ενός φαρμάκου, για παράδειγμα για την θεραπευτική αγωγή μιας κληρονομικής ασθένειας.
CY20161100215T 2000-09-21 2016-03-11 Επαγωγη παρακαμψης εξωνιου σε ευκαριωτικα κυτταρα CY1117522T1 (el)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
EP00203283A EP1191097A1 (en) 2000-09-21 2000-09-21 Induction of exon skipping in eukaryotic cells
EP12198485.0A EP2594641B1 (en) 2000-09-21 2001-09-21 Induction of exon skipping in eukaryotic cells

Publications (1)

Publication Number Publication Date
CY1117522T1 true CY1117522T1 (el) 2017-04-26

Family

ID=8172043

Family Applications (10)

Application Number Title Priority Date Filing Date
CY20081101494T CY1109601T1 (el) 2000-09-21 2008-12-23 Επαγωγη παρακαμψης εξωνιου σε ευκαριωτικα κυτταρα
CY20161100207T CY1117521T1 (el) 2000-09-21 2016-03-10 Επαγωγη παρακαμψης εξωνιου σε ευκαριωτικα κυτταρα
CY20161100216T CY1117318T1 (el) 2000-09-21 2016-03-11 Επαγωγη παρακαμψης εξωνιου σε ευκαριωτικα κυτταρα
CY20161100215T CY1117522T1 (el) 2000-09-21 2016-03-11 Επαγωγη παρακαμψης εξωνιου σε ευκαριωτικα κυτταρα
CY20161100443T CY1117887T1 (el) 2000-09-21 2016-05-23 Επαγωγη παρακαμψης εξωνιου σε ευκαριωτικα κυτταρα
CY20161100724T CY1117852T1 (el) 2000-09-21 2016-07-22 Επαγωγη παρακαμψης εξωνιου σε ευκαριωτικα κυτταρα
CY20171100302T CY1118972T1 (el) 2000-09-21 2017-03-09 Επαγωγη παρακαμψης εξωνιου σε ευκαριωτικα κυτταρα
CY20171100691T CY1119026T1 (el) 2000-09-21 2017-06-29 Επαγωγη παρακαμψης εξωνιου σε ευκαριωτικα κυτταρα
CY20171100750T CY1119088T1 (el) 2000-09-21 2017-07-14 Επαγωγη παρακαμψης εξωνιου σε ευκαριωτικα κυτταρα
CY20181100969T CY1121139T1 (el) 2000-09-21 2018-09-19 Επαγωγη παρακαμψης εξωνιου σε ευκαριωτικα κυτταρα

Family Applications Before (3)

Application Number Title Priority Date Filing Date
CY20081101494T CY1109601T1 (el) 2000-09-21 2008-12-23 Επαγωγη παρακαμψης εξωνιου σε ευκαριωτικα κυτταρα
CY20161100207T CY1117521T1 (el) 2000-09-21 2016-03-10 Επαγωγη παρακαμψης εξωνιου σε ευκαριωτικα κυτταρα
CY20161100216T CY1117318T1 (el) 2000-09-21 2016-03-11 Επαγωγη παρακαμψης εξωνιου σε ευκαριωτικα κυτταρα

Family Applications After (6)

Application Number Title Priority Date Filing Date
CY20161100443T CY1117887T1 (el) 2000-09-21 2016-05-23 Επαγωγη παρακαμψης εξωνιου σε ευκαριωτικα κυτταρα
CY20161100724T CY1117852T1 (el) 2000-09-21 2016-07-22 Επαγωγη παρακαμψης εξωνιου σε ευκαριωτικα κυτταρα
CY20171100302T CY1118972T1 (el) 2000-09-21 2017-03-09 Επαγωγη παρακαμψης εξωνιου σε ευκαριωτικα κυτταρα
CY20171100691T CY1119026T1 (el) 2000-09-21 2017-06-29 Επαγωγη παρακαμψης εξωνιου σε ευκαριωτικα κυτταρα
CY20171100750T CY1119088T1 (el) 2000-09-21 2017-07-14 Επαγωγη παρακαμψης εξωνιου σε ευκαριωτικα κυτταρα
CY20181100969T CY1121139T1 (el) 2000-09-21 2018-09-19 Επαγωγη παρακαμψης εξωνιου σε ευκαριωτικα κυτταρα

Country Status (17)

Country Link
US (6) US7973015B2 (el)
EP (14) EP1191097A1 (el)
JP (7) JP4846965B2 (el)
AT (1) ATE409224T2 (el)
AU (4) AU2002211062C1 (el)
CA (1) CA2423044C (el)
CY (10) CY1109601T1 (el)
DE (1) DE60135936D1 (el)
DK (10) DK2801618T3 (el)
ES (11) ES2581285T3 (el)
HK (8) HK1184818A1 (el)
LT (3) LT2940139T (el)
NZ (1) NZ524853A (el)
PT (7) PT2636741E (el)
SI (1) SI2801618T1 (el)
TR (1) TR201810606T4 (el)
WO (1) WO2002024906A1 (el)

Families Citing this family (122)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2001257526B2 (en) 2000-05-04 2007-07-19 Avi Biopharma, Inc. Splice-region antisense composition and method
EP1191097A1 (en) 2000-09-21 2002-03-27 Leids Universitair Medisch Centrum Induction of exon skipping in eukaryotic cells
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.
EP2530156B1 (en) * 2002-11-25 2015-11-18 Masafumi Matsuo ENA nucleic acid drugs modifying splicing in mRNA precursor
AU2003225410A1 (en) 2003-03-21 2004-10-11 Academisch Ziekenhuis Leiden Modulation of exon recognition in pre-mrna by interfering with the secondary rna structure
DE602004028930D1 (de) * 2003-04-29 2010-10-14 Avi Biopharma Inc Zusammensetzungen zur verbesserung der antisense-wirksamkeit und des transports von nukleinsäureanalog in zellen
US20050288246A1 (en) * 2004-05-24 2005-12-29 Iversen Patrick L Peptide conjugated, inosine-substituted antisense oligomer compound and method
USRE48960E1 (en) 2004-06-28 2022-03-08 The University Of Western Australia Antisense oligonucleotides for inducing exon skipping and methods of use thereof
SI1766010T1 (sl) 2004-06-28 2011-06-30 Univ Western Australia Protismiselni oligonukleotidi za induciranje preskakovanja eksonov in postopki za njihovo uporabo
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
EP1855694B1 (en) 2005-02-09 2020-12-02 Sarepta Therapeutics, Inc. Antisense composition for treating muscle atrophy
NZ563206A (en) * 2005-04-22 2009-09-25 Academisch Ziekenhuis Leiden Modulation of exon recognition in pre-mRNA by interfering with the binding or SR proteins and by interfering with secondary RNA structure
US8067571B2 (en) 2005-07-13 2011-11-29 Avi Biopharma, Inc. Antibacterial antisense oligonucleotide and method
US7785834B2 (en) * 2005-11-10 2010-08-31 Ercole Biotech, Inc. Soluble TNF receptors and their use in treatment of disease
WO2007058894A2 (en) * 2005-11-10 2007-05-24 The University Of North Carolina At Chapel Hill Splice switching oligomers for tnf superfamily receptors and their use in treatment of disease
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
US20090264353A1 (en) * 2007-10-19 2009-10-22 Santaris Pharma A/S Splice Switching Oligomers for TNF Superfamily Receptors and their Use in Treatment of Disease
AU2008271050B2 (en) * 2007-06-29 2014-11-06 Sarepta Therapeutics, Inc. Tissue specific peptide conjugates and methods
US20100016215A1 (en) 2007-06-29 2010-01-21 Avi Biopharma, Inc. Compound and method for treating myotonic dystrophy
JP2010533170A (ja) 2007-07-12 2010-10-21 プロセンサ テクノロジーズ ビー．ブイ． 化合物を種々の選択された臓器、組織又は腫瘍細胞に標的化するための分子
DK2167136T3 (en) 2007-07-12 2016-07-25 Biomarin Tech Bv Molecules for targeting compounds at different selected organs or tissues
USRE48468E1 (en) 2007-10-26 2021-03-16 Biomarin Technologies B.V. Means and methods for counteracting muscle disorders
CN101896186A (zh) 2007-10-26 2010-11-24 莱顿教学医院 对抗肌肉病症的方式和方法
JP2011510678A (ja) 2008-02-08 2011-04-07 プロセンサ ホールディング ビーブイ Ｄｎａ反復不安定性関連遺伝性障害を治療するための方法及び手段
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
US8084601B2 (en) 2008-09-11 2011-12-27 Royal Holloway And Bedford New College Royal Holloway, University Of London Oligomers
EP3133160B1 (en) 2008-10-24 2018-12-12 Sarepta Therapeutics, Inc. Exon skipping compositions for dmd
EP2370451B1 (en) 2008-12-02 2016-11-16 Wave Life Sciences Japan, Inc. Method for the synthesis of phosphorus atom modified nucleic acids
US20120046342A1 (en) 2009-04-24 2012-02-23 Prosensa Technologies B.V. Oligonucleotide comprising an inosine for treating dmd
US20110269665A1 (en) 2009-06-26 2011-11-03 Avi Biopharma, Inc. Compound and method for treating myotonic dystrophy
SG177564A1 (en) 2009-07-06 2012-02-28 Ontorii Inc Novel nucleic acid prodrugs and methods of use thereof
US20120270930A1 (en) 2009-10-29 2012-10-25 Academisch Ziekenhuis Leiden H.O.D.N. Lumc Methods and compositions for dysferlin exon-skipping
BR122023023194A2 (pt) 2009-11-12 2023-12-26 The University Of Western Australia Oligonucleotídeo antissentido, composição e respectivo uso
JP6141018B2 (ja) 2009-12-24 2017-06-07 バイオマリン テクノロジーズ ベー．フェー． 炎症性障害を治療するための分子
DK2601294T3 (en) 2010-08-05 2019-02-18 Academisch Ziekenhuis Leiden ANTISENSE OLIGONUCLEOTIDE CALCULATED FOR THE REMOVAL OF PROTEOLYTIC DIVISION PLACES FROM PROTEINS
TWI541024B (zh) 2010-09-01 2016-07-11 日本新藥股份有限公司 反義核酸
JP5868324B2 (ja) 2010-09-24 2016-02-24 株式会社Ｗａｖｅ Ｌｉｆｅ Ｓｃｉｅｎｃｅｓ Ｊａｐａｎ 不斉補助基
CA2831827A1 (en) 2011-04-05 2012-10-11 Academisch Ziekenhuis Leiden H.O.D.N. Lumc Compounds and methods for altering activin receptor-like kinase signalling
US9161948B2 (en) 2011-05-05 2015-10-20 Sarepta Therapeutics, Inc. Peptide oligonucleotide conjugates
RU2014105311A (ru) 2011-07-19 2015-08-27 Уэйв Лайф Сайенсес Пте. Лтд. Способы синтеза функционализованных нуклеиновых кислот
WO2013033407A2 (en) 2011-08-30 2013-03-07 The Regents Of The University Of California Identification of small molecules that enhance therapeutic exon skipping
CA2847664C (en) 2011-09-05 2021-08-31 Stichting Katholieke Universiteit Antisense oligonucleotides for the treatment of leber congenital amaurosis
US20130085139A1 (en) 2011-10-04 2013-04-04 Royal Holloway And Bedford New College Oligomers
CN108588073A (zh) * 2011-12-28 2018-09-28 日本新药株式会社 反义核酸
AU2013212758A1 (en) 2012-01-27 2014-08-14 Biomarin Technologies B.V. RNA modulating oligonucleotides with improved characteristics for the treatment of Duchenne and Becker muscular dystrophy
DE102012103041A1 (de) 2012-04-10 2013-10-10 Eberhard-Karls-Universität Tübingen Universitätsklinikum Behandlung der dilativen Kardiomyopathie mittels Antisense-Oligonucleotiden
WO2014007620A2 (en) * 2012-07-03 2014-01-09 Prosensa Technologies B.V. Oligonucleotide for the treatment of muscular dystrophy patients
SG11201500243WA (en) 2012-07-13 2015-04-29 Shin Nippon Biomedical Lab Ltd Chiral nucleic acid adjuvant
SG11201500239VA (en) 2012-07-13 2015-03-30 Wave Life Sciences Japan Asymmetric auxiliary group
US9982257B2 (en) 2012-07-13 2018-05-29 Wave Life Sciences Ltd. Chiral control
PL2880053T3 (pl) 2012-08-01 2021-01-11 Ikaika Therapeutics, Llc Łagodzenie uszkodzenia tkanek i włóknienia przez przeciwciało przeciwko ltbp4
NZ775701A (en) 2013-03-14 2022-08-26 Sarepta Therapeutics Inc Exon skipping compositions for treating muscular dystrophy
EP2968991A2 (en) 2013-03-15 2016-01-20 Sarepta Therapeutics, Inc. Improved compositions for treating muscular dystrophy
US9849066B2 (en) 2013-04-24 2017-12-26 Corning Incorporated Delamination resistant pharmaceutical glass containers containing active pharmaceutical ingredients
JPWO2015108047A1 (ja) 2014-01-15 2017-03-23 株式会社新日本科学 免疫誘導活性を有するキラル核酸アジュバンド及び免疫誘導活性剤
JPWO2015108046A1 (ja) 2014-01-15 2017-03-23 株式会社新日本科学 抗アレルギー作用を有するキラル核酸アジュバンド及び抗アレルギー剤
US10149905B2 (en) 2014-01-15 2018-12-11 Shin Nippon Biomedical Laboratories, Ltd. Chiral nucleic acid adjuvant having antitumor effect and antitumor agent
DK3094728T3 (da) 2014-01-16 2022-05-16 Wave Life Sciences Ltd Kiralt design
EP3097188B1 (en) 2014-01-24 2018-08-29 AM-Pharma B.V. Downstream processing of an alkaline phosphatase
US9926544B2 (en) 2014-01-24 2018-03-27 Am-Pharma B.V. Chimeric alkaline phosphatase-like proteins
TR201901939T4 (tr) 2014-03-12 2019-03-21 Nat Center Neurology & Psychiatry Antisens nükleik asit.
WO2015171918A2 (en) 2014-05-07 2015-11-12 Louisiana State University And Agricultural And Mechanical College Compositions and uses for treatment thereof
AU2015286663B2 (en) 2014-07-10 2021-09-23 Stichting Radboud Universitair Medisch Centrum Antisense oligonucleotides for the treatment of usher syndrome type 2
US20160058889A1 (en) * 2014-08-11 2016-03-03 The Board Of Regents Of The University Of Texas System Prevention of muscular dystrophy by crispr/cas9-mediated gene editing
WO2016037165A1 (en) 2014-09-07 2016-03-10 Selecta Biosciences, Inc. Methods and compositions for attenuating exon skipping anti-viral transfer vector immune responses
US20170266320A1 (en) * 2014-12-01 2017-09-21 President And Fellows Of Harvard College RNA-Guided Systems for In Vivo Gene Editing
GB201504124D0 (en) 2015-03-11 2015-04-22 Proqr Therapeutics B V Oligonucleotides
MA41795A (fr) 2015-03-18 2018-01-23 Sarepta Therapeutics Inc Exclusion d'un exon induite par des composés antisens dans la myostatine
WO2016196897A1 (en) 2015-06-04 2016-12-08 Sarepta Therapeutics, Inc. Methods and compounds for treatment of lymphocyte-related diseases and conditions
LT3351633T (lt) 2015-09-15 2020-08-10 Nippon Shinyaku Co., Ltd. Priešprasmė nukleorūgštis
WO2017062835A2 (en) 2015-10-09 2017-04-13 Sarepta Therapeutics, Inc. Compositions and methods for treating duchenne muscular dystrophy and related disorders
IL258230B (en) 2015-10-09 2022-09-01 Wave Life Sciences Ltd Oligonucleotide preparations and methods thereof
WO2017136435A1 (en) 2016-02-01 2017-08-10 The Usa, As Represented By The Secretary, Department Of Health And Human Services Office Of Technology Transfer National Institute Of Health Compounds for modulating fc-epsilon-ri-beta expression and uses thereof
AU2017257292A1 (en) 2016-04-25 2018-12-06 Proqr Therapeutics Ii B.V. Oligonucleotides to treat eye disease
KR20190024977A (ko) 2016-06-30 2019-03-08 사렙타 쎄러퓨틱스 인코퍼레이티드 근육 이상증에 대한 엑손 스킵핑 올리고머
SG10201607303YA (en) * 2016-09-01 2018-04-27 Agency Science Tech & Res Antisense oligonucleotides to induce exon skipping
GB201616202D0 (en) 2016-09-23 2016-11-09 Proqr Therapeutics Ii Bv Antisense oligonucleotides for the treatment of eye deisease
WO2018109011A1 (en) 2016-12-13 2018-06-21 Stichting Katholieke Universiteit Antisense oligonucleotides for the treatment of stargardt disease
IL297528A (en) 2016-12-19 2022-12-01 Sarepta Therapeutics Inc Exon-skipping oligomer conjugates for muscular dystrophy
WO2018118599A1 (en) 2016-12-19 2018-06-28 Sarepta Therapeutics, Inc. Exon skipping oligomer conjugates for muscular dystrophy
BR112019012651A2 (pt) 2016-12-19 2020-01-28 Sarepta Therapeutics Inc conjugados de oligômero de salto de éxon para distrofia muscular
CA3070242A1 (en) 2017-07-18 2019-01-24 Calimmune, Inc. Compositions and methods for treating beta-hemoglobinopathies
GB201711809D0 (en) * 2017-07-21 2017-09-06 Governors Of The Univ Of Alberta Antisense oligonucleotide
RU2020105929A (ru) 2017-08-04 2021-09-06 Скайхоук Терапьютикс, Инк. Способы и композиции для модулирования сплайсинга
CN111201325A (zh) 2017-08-11 2020-05-26 新加坡科技研究局 用于筛选剪接变异体或事件的方法
EA201991450A1 (ru) 2017-09-22 2019-12-30 Сарепта Терапьютикс, Инк. Конъюгаты олигомеров для пропуска экзона при мышечной дистрофии
JP2020536060A (ja) 2017-09-28 2020-12-10 サレプタ セラピューティクス， インコーポレイテッド 筋ジストロフィーを処置するための併用療法
CN111542336A (zh) 2017-10-13 2020-08-14 西莱克塔生物科技公司 用于减弱抗病毒转移载体igm应答的方法和组合物
GB201803010D0 (en) 2018-02-26 2018-04-11 Royal Holloway & Bedford New College Neurodegenerative disorders
US10758629B2 (en) 2018-05-29 2020-09-01 Sarepta Therapeutics, Inc. Exon skipping oligomer conjugates for muscular dystrophy
WO2020015959A1 (en) 2018-07-19 2020-01-23 Stichting Katholieke Universiteit Antisense oligonucleotides rescue aberrant splicing of abca4.
CN112955153A (zh) 2018-08-02 2021-06-11 达因疗法公司 肌肉靶向复合物及其用于治疗肌养蛋白病的用途
US11168141B2 (en) 2018-08-02 2021-11-09 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
EP3830259A4 (en) 2018-08-02 2022-05-04 Dyne Therapeutics, Inc. MUSCLE TARGETING COMPLEXES AND THEIR USES FOR THE TREATMENT OF FACIOSCAPULOHUMERAL MUSCULAR DYSTROPHY
US20220049250A1 (en) 2018-12-04 2022-02-17 Stichting Katholieke Universiteit Antisense Oligonucleotides Rescue Aberrant Splicing of ABCA4
AU2019417697A1 (en) 2018-12-23 2021-07-08 Csl Behring L.L.C. Haematopoietic stem cell-gene therapy for wiskott-aldrich syndrome
EP3897831A1 (en) 2018-12-23 2021-10-27 CSL Behring LLC Donor t-cells with kill switch
WO2020148400A1 (en) 2019-01-16 2020-07-23 Stichting Katholieke Universiteit Antisense oligonucleotides for use in the treatment of crpc
AU2020214704A1 (en) 2019-01-28 2021-08-26 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of leber's congenital amaurosis
CN114007613A (zh) 2019-02-05 2022-02-01 斯基霍克疗法公司 用于调节剪接的方法和组合物
US20220177894A1 (en) 2019-04-02 2022-06-09 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for immunotherapy
AU2020259856A1 (en) 2019-04-18 2021-11-18 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of usher syndrome
EP3976802A1 (en) 2019-05-28 2022-04-06 Selecta Biosciences, Inc. Methods and compositions for attenuated anti-viral transfer vector immune response
WO2020254249A1 (en) 2019-06-21 2020-12-24 Proqr Therapeutics Ii B.V. Delivery of nucleic acids for the treatment of auditory disorders
US20220290154A1 (en) 2019-08-08 2022-09-15 Stichting Radboud Universitair Medisch Centrum Antisense oligonucleotides rescue aberrant splicing of ABCA4
EP4051794A1 (en) 2019-10-31 2022-09-07 Stichting Radboud universitair medisch centrum Allele-specific silencing therapy for dfna9 using antisense oligonucleotides
CA3173049A1 (en) 2020-02-28 2021-09-02 Nippon Shinyaku Co., Ltd. Antisense nucleic acid inducing skipping of exon 51
EP4114945A1 (en) 2020-03-04 2023-01-11 ProQR Therapeutics II B.V. Antisense oligonucleotides for use in the treatment of usher syndrome
CN115997015A (zh) 2020-06-26 2023-04-21 美国杰特贝林生物制品有限公司 具有杀伤开关的供体t细胞
WO2022090256A1 (en) 2020-10-26 2022-05-05 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of stargardt disease
CN112430645A (zh) * 2020-12-09 2021-03-02 北京华瑞康源生物科技发展有限公司 一种多重实时荧光pcr法检测人dmd基因拷贝数的相对定量方法及试剂盒
CA3218805A1 (en) 2021-05-10 2022-11-17 Ziqing QIAN Compositions and methods for intracellular therapeutics
CA3222824A1 (en) 2021-06-23 2022-12-29 Entrada Therapeutics, Inc. Antisense compounds and methods for targeting cug repeats
WO2022269016A1 (en) 2021-06-25 2022-12-29 Stichting Radboud Universitair Medisch Centrum Allele-specific silencing therapy for dfna21 using antisense oligonucleotides
US11771776B2 (en) 2021-07-09 2023-10-03 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
US11638761B2 (en) 2021-07-09 2023-05-02 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating Facioscapulohumeral muscular dystrophy
US20230141563A1 (en) 2021-10-12 2023-05-11 Selecta Biosciences, Inc. Methods and compositions for attenuating anti-viral transfer vector igm responses
EP4215614A1 (en) 2022-01-24 2023-07-26 Dynacure Combination therapy for dystrophin-related diseases
US20230357437A1 (en) 2022-03-09 2023-11-09 Selecta Biosciences, Inc. Immunosuppressants in combination with anti-igm agents and related dosing
DE102022124232A1 (de) 2022-09-21 2024-03-21 Carl von Ossietzky Universität Oldenburg, Körperschaft des öffentlichen Rechts Antisense-Oligonukleotide für die Behandlung des Joubert-Syndroms

Family Cites Families (64)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5034506A (en) * 1985-03-15 1991-07-23 Anti-Gene Development Group Uncharged morpholino-based polymers having achiral intersubunit linkages
US5541308A (en) * 1986-11-24 1996-07-30 Gen-Probe Incorporated Nucleic acid probes for detection and/or quantitation of non-viral organisms
US5766847A (en) * 1988-10-11 1998-06-16 Max-Planck-Gesellschaft Zur Forderung Der Wissenschaften E.V. Process for analyzing length polymorphisms in DNA regions
US6867195B1 (en) * 1989-03-21 2005-03-15 Vical Incorporated Lipid-mediated polynucleotide administration to reduce likelihood of subject's becoming infected
US5608046A (en) * 1990-07-27 1997-03-04 Isis Pharmaceuticals, Inc. Conjugated 4'-desmethyl nucleoside analog compounds
FR2675803B1 (fr) * 1991-04-25 1996-09-06 Genset Sa Oligonucleotides fermes, antisens et sens et leurs applications.
EP0558697A1 (en) 1991-06-28 1993-09-08 Massachusetts Institute Of Technology Localized oligonucleotide therapy
JPH07501204A (ja) 1991-06-28 1995-02-09 マサチューセッツ インスティテュート オブ テクノロジー 局所的オリゴヌクレオチド療法
US6200747B1 (en) * 1992-01-28 2001-03-13 North Shore University Hospital Research Corp. Method and kits for detection of fragile X specific, GC-rich DNA sequences
US5869252A (en) † 1992-03-31 1999-02-09 Abbott Laboratories Method of multiplex ligase chain reaction
US6172208B1 (en) * 1992-07-06 2001-01-09 Genzyme Corporation Oligonucleotides modified with conjugate groups
US5418139A (en) * 1993-02-10 1995-05-23 University Of Iowa Research Foundation Method for screening for cardiomyopathy
AU6786594A (en) * 1993-05-11 1994-12-12 University Of North Carolina At Chapel Hill, The Antisense oligonucleotides which combat aberrant splicing and methods of using the same
US5741645A (en) * 1993-06-29 1998-04-21 Regents Of The University Of Minnesota Gene sequence for spinocerebellar ataxia type 1 and method for diagnosis
US5624803A (en) * 1993-10-14 1997-04-29 The Regents Of The University Of California In vivo oligonucleotide generator, and methods of testing the binding affinity of triplex forming oligonucleotides derived therefrom
US5627263A (en) * 1993-11-24 1997-05-06 La Jolla Cancer Research Foundation Integrin-binding peptides
US5962332A (en) * 1994-03-17 1999-10-05 University Of Massachusetts Detection of trinucleotide repeats by in situ hybridization
US5968909A (en) 1995-08-04 1999-10-19 Hybridon, Inc. Method of modulating gene expression with reduced immunostimulatory response
US5854223A (en) 1995-10-06 1998-12-29 The Trustees Of Columbia University In The City Of New York S-DC28 as an antirestenosis agent after balloon injury
US7034009B2 (en) * 1995-10-26 2006-04-25 Sirna Therapeutics, Inc. Enzymatic nucleic acid-mediated treatment of ocular diseases or conditions related to levels of vascular endothelial growth factor receptor (VEGF-R)
US6300060B1 (en) * 1995-11-09 2001-10-09 Dana-Farber Cancer Institute, Inc. Method for predicting the risk of prostate cancer morbidity and mortality
BR9707529A (pt) 1996-02-14 2000-01-04 Isis Pharmaceuticals Inc Oligunucleotìdeo especificamente hibridizável com dna ou rna.
US6251589B1 (en) * 1996-07-18 2001-06-26 Srl, Inc. Method for diagnosing spinocerebellar ataxia type 2 and primers therefor
US5853995A (en) * 1997-01-07 1998-12-29 Research Development Foundation Large scale genotyping of diseases and a diagnostic test for spinocerebellar ataxia type 6
US20020137890A1 (en) * 1997-03-31 2002-09-26 Genentech, Inc. Secreted and transmembrane polypeptides and nucleic acids encoding the same
US6329501B1 (en) * 1997-05-29 2001-12-11 Auburn University Methods and compositions for targeting compounds to muscle
US6514755B1 (en) * 1998-08-18 2003-02-04 Regents Of The University Of Minnesota SCA7 gene and methods of use
US6280938B1 (en) * 1997-08-19 2001-08-28 Regents Of The University Of Minnesota SCA7 gene and method of use
US6794499B2 (en) * 1997-09-12 2004-09-21 Exiqon A/S Oligonucleotide analogues
US6130207A (en) * 1997-11-05 2000-10-10 South Alabama Medical Science Foundation Cell-specific molecule and method for importing DNA into a nucleus
JP3012923B2 (ja) * 1998-01-26 2000-02-28 新潟大学長 Ｃａｇリピート病の治療薬
KR100280219B1 (ko) * 1998-02-26 2001-04-02 이수빈 삼핵산 반복 서열을 이용한 신경정신 질환의 진단 방법 및 진단 시약
US6322978B1 (en) * 1998-04-20 2001-11-27 Joslin Diabetes Center, Inc. Repeat polymorphism in the frataxin gene and uses therefore
CA2334960C (en) * 1998-06-10 2012-01-03 Biognostik Gesellschaft Fur Biomolekulare Diagnostik Mbh Combination of tgf-.beta. inhibition and immune stimulation to treat hyperproliferative diseases
US20030096955A1 (en) * 1998-09-01 2003-05-22 Genentech, Inc. Secreted and transmembrane polypeptides and nucleic acids encoding the same
IL142828A0 (en) * 1998-09-25 2002-03-10 Childrens Medical Center Short peptides which selectively modulate the activity of protein kinases
US6210892B1 (en) * 1998-10-07 2001-04-03 Isis Pharmaceuticals, Inc. Alteration of cellular behavior by antisense modulation of mRNA processing
US6172216B1 (en) 1998-10-07 2001-01-09 Isis Pharmaceuticals Inc. Antisense modulation of BCL-X expression
JP2000125448A (ja) 1998-10-14 2000-04-28 Yazaki Corp 電気接続箱
US6399575B1 (en) * 1998-11-10 2002-06-04 Auburn University Methods and compositions for targeting compounds to the central nervous system
US6133031A (en) * 1999-08-19 2000-10-17 Isis Pharmaceuticals Inc. Antisense inhibition of focal adhesion kinase expression
US20040226056A1 (en) * 1998-12-22 2004-11-11 Myriad Genetics, Incorporated Compositions and methods for treating neurological disorders and diseases
JP2000256547A (ja) 1999-03-10 2000-09-19 Sumitomo Dow Ltd 耐熱性プラスチックカード用樹脂組成物
US20020049173A1 (en) 1999-03-26 2002-04-25 Bennett C. Frank Alteration of cellular behavior by antisense modulation of mRNA processing
US6379698B1 (en) * 1999-04-06 2002-04-30 Isis Pharmaceuticals, Inc. Fusogenic lipids and vesicles
JP2000325085A (ja) 1999-05-21 2000-11-28 Masafumi Matsuo デュシェンヌ型筋ジストロフィー治療剤
US20030236214A1 (en) * 1999-06-09 2003-12-25 Wolff Jon A. Charge reversal of polyion complexes and treatment of peripheral occlusive disease
AU5625500A (en) * 1999-06-18 2001-01-09 Emory University Huntington disease cellular model: stably transfected pc12 cells expressing mutant huntingtin
EP1133993A1 (en) 2000-03-10 2001-09-19 Max-Planck-Gesellschaft zur Förderung der Wissenschaften e.V. Substances for the treatment of spinal muscular atrophy
US6653467B1 (en) 2000-04-26 2003-11-25 Jcr Pharmaceutical Co., Ltd. Medicament for treatment of Duchenne muscular dystrophy
WO2001085751A1 (en) * 2000-05-09 2001-11-15 Reliable Biopharmaceutical, Inc. Polymeric compounds useful as prodrugs
US20030124523A1 (en) * 2000-06-22 2003-07-03 Asselbergs Fredericus Alphonsus Maria Organic compounds
US6794192B2 (en) * 2000-06-29 2004-09-21 Pfizer Inc. Target
US6727355B2 (en) 2000-08-25 2004-04-27 Jcr Pharmaceuticals Co., Ltd. Pharmaceutical composition for treatment of Duchenne muscular dystrophy
JP4836366B2 (ja) * 2000-08-25 2011-12-14 雅文 松尾 デュシェンヌ型筋ジストロフィー治療剤
EP1191097A1 (en) 2000-09-21 2002-03-27 Leids Universitair Medisch Centrum Induction of exon skipping in eukaryotic cells
JP3995996B2 (ja) * 2002-06-21 2007-10-24 エスアイアイ・プリンテック株式会社 インクジェットヘッド及びインクジェット式記録装置
AU2003225410A1 (en) 2003-03-21 2004-10-11 Academisch Ziekenhuis Leiden Modulation of exon recognition in pre-mrna by interfering with the secondary rna structure
SI1766010T1 (sl) * 2004-06-28 2011-06-30 Univ Western Australia Protismiselni oligonukleotidi za induciranje preskakovanja eksonov in postopki za njihovo uporabo
CN101896186A (zh) * 2007-10-26 2010-11-24 莱顿教学医院 对抗肌肉病症的方式和方法
US8084601B2 (en) * 2008-09-11 2011-12-27 Royal Holloway And Bedford New College Royal Holloway, University Of London Oligomers
JP6141018B2 (ja) * 2009-12-24 2017-06-07 バイオマリン テクノロジーズ ベー．フェー． 炎症性障害を治療するための分子
AU2013212758A1 (en) * 2012-01-27 2014-08-14 Biomarin Technologies B.V. RNA modulating oligonucleotides with improved characteristics for the treatment of Duchenne and Becker muscular dystrophy
JP5794194B2 (ja) † 2012-04-19 2015-10-14 東京エレクトロン株式会社 基板処理装置

Also Published As

Publication number Publication date
CY1109601T1 (el) 2014-08-13
HK1216906A1 (zh) 2016-12-09
US20090228998A1 (en) 2009-09-10
EP2636741B1 (en) 2016-04-27
EP2284264B1 (en) 2016-12-14
PT2594640E (pt) 2016-03-11
LT2801618T (lt) 2017-09-11
EP2594640B1 (en) 2015-12-30
LT2284264T (lt) 2017-03-10
CY1121139T1 (el) 2020-05-29
ES2315788T3 (es) 2009-04-01
DK2636742T3 (en) 2017-07-24
EP2940139A1 (en) 2015-11-04
EP2602322B1 (en) 2016-03-02
US20150322434A1 (en) 2015-11-12
ES2690049T3 (es) 2018-11-19
EP2801618A1 (en) 2014-11-12
ES2561293T3 (es) 2016-02-25
AU2011201325A1 (en) 2011-04-14
DK2594642T3 (en) 2016-03-21
WO2002024906A1 (en) 2002-03-28
HK1184821A1 (zh) 2014-01-30
EP2801618B1 (en) 2017-04-19
PT2940139T (pt) 2018-10-15
ES2567417T3 (es) 2016-04-22
EP1619249A1 (en) 2006-01-25
CY1119026T1 (el) 2018-01-10
PT2284264T (pt) 2017-03-06
JP2017200952A (ja) 2017-11-09
EP2636742B1 (en) 2017-04-19
EP2594640A1 (en) 2013-05-22
US20140350076A1 (en) 2014-11-27
HK1184818A1 (zh) 2014-01-30
PT2636742T (pt) 2017-06-02
ES2610568T3 (es) 2017-04-28
ES2561292T3 (es) 2016-02-25
DK2594641T3 (en) 2016-03-21
EP1619249B2 (en) 2020-01-08
EP2594641A1 (en) 2013-05-22
JP2004509622A (ja) 2004-04-02
HK1188249A1 (zh) 2014-04-25
PT2801618T (pt) 2017-07-25
DK2801618T3 (en) 2017-07-24
DK1619249T3 (da) 2009-02-02
ES2561294T3 (es) 2016-02-25
AU2007234488A1 (en) 2007-12-06
JP6511124B2 (ja) 2019-05-15
PT2636741E (pt) 2016-06-09
DE60135936D1 (de) 2008-11-06
ES2609421T3 (es) 2017-04-20
CA2423044A1 (en) 2002-03-28
ES2629747T3 (es) 2017-08-14
EP1320597A2 (en) 2003-06-25
JP6126983B2 (ja) 2017-05-10
EP2602322A1 (en) 2013-06-12
ES2628349T3 (es) 2017-08-02
CY1119088T1 (el) 2018-01-10
EP1191097A1 (en) 2002-03-27
JP6250078B2 (ja) 2017-12-20
JP2018052981A (ja) 2018-04-05
AU2002211062C1 (en) 2011-02-03
EP1619249B1 (en) 2008-09-24
EP2636740B1 (en) 2016-11-09
DK2284264T3 (en) 2017-03-13
DK2636741T3 (en) 2016-07-04
NZ524853A (en) 2004-11-26
EP2940139B1 (en) 2018-07-04
CA2423044C (en) 2016-08-23
AU1106202A (en) 2002-04-02
WO2002024906A9 (en) 2003-01-23
JP4846965B2 (ja) 2011-12-28
AU2002211062B2 (en) 2007-11-29
DK2940139T3 (en) 2018-10-08
CY1117318T1 (el) 2017-04-26
DK1619249T4 (da) 2020-04-06
US20030235845A1 (en) 2003-12-25
AU2011201325B2 (en) 2011-12-22
JP2016104795A (ja) 2016-06-09
HK1184820A1 (zh) 2014-01-30
JP6425775B2 (ja) 2018-11-21
EP2284264A1 (en) 2011-02-16
EP2636741A1 (en) 2013-09-11
CY1117852T1 (el) 2017-05-17
ATE409224T2 (de) 2008-10-15
US20080209581A1 (en) 2008-08-28
AU2007234488B2 (en) 2011-02-17
CY1117521T1 (el) 2017-04-26
TR201810606T4 (tr) 2018-08-27
PT1619249E (pt) 2009-01-07
JP2019073555A (ja) 2019-05-16
DK2594640T3 (en) 2016-03-21
EP2594642A1 (en) 2013-05-22
US7973015B2 (en) 2011-07-05
EP2594642B1 (en) 2015-12-30
US20150361424A1 (en) 2015-12-17
JP2011200235A (ja) 2011-10-13
CY1117887T1 (el) 2017-05-17
LT2940139T (lt) 2018-09-10
EP2594641B1 (en) 2015-12-30
EP2636742A1 (en) 2013-09-11
HK1184819A1 (zh) 2014-01-30
JP2014065733A (ja) 2014-04-17
HK1188250A1 (zh) 2014-04-25
CY1118972T1 (el) 2018-01-10
EP2636740A1 (en) 2013-09-11
EP3382021A1 (en) 2018-10-03
ES2581285T3 (es) 2016-09-05
ES2315788T5 (es) 2020-09-17
SI2801618T1 (sl) 2017-07-31
DK2602322T3 (en) 2016-04-18
HK1203554A1 (en) 2015-10-30

Similar Documents

Publication Publication Date Title
CY1119088T1 (el) Επαγωγη παρακαμψης εξωνιου σε ευκαριωτικα κυτταρα
IL193373A (en) Dna sequences comprising gene transcription regulatory qualities and methods for detecting and using such dna sequences
DE60111620D1 (de) Aktiviertes protein c zur behandlung von pankreatitis
DE50209165D1 (de) Verfahren zur herstellung von d-pantothensäure und/oder salze als zusatz zu tierfuttermitteln
DE60001683D1 (de) Verfahren zur Reinigung von 3,7,11,15-Tetramethyl-2,4,6,10,14-hexadecapentaenonsäure
ATE455089T1 (de) Verfahren zur herstellung von aminomethylcycloalkan-essigsäuren
ATE396262T1 (de) Rna polymerase i transkriptionsfaktor tif-ia
DE60225338D1 (de) Verfahren zur herstellung von derivaten von 3,3-dimethyl-2-formylcyclopropanekarbonsäure
FR2860733B1 (fr) Methode de pretraitement de catalyseurs a base d'acide phosphorique
DE50306247D1 (de) Verfahren zur Herstellung von 2-Phenyl-benzimidazol-5-sulfonsäure aus 3,4-Diaminobenzol-sulfonsäure
DE60218359D1 (de) Verfahren zur herstellung von 2,4,4,6-tetrabrom-2,5-cyclohexadienon
WO2004026905A3 (en) Transporter/channel proteins