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PL2320952T3 - ULEPSZONE CZĄSTECZKI CZĄSTECZKI TRANS-SPLICINGUJĄCEJ PRE-mRNA (RTM) I ICH ZASTOSOWANIE - Google Patents

ULEPSZONE CZĄSTECZKI CZĄSTECZKI TRANS-SPLICINGUJĄCEJ PRE-mRNA (RTM) I ICH ZASTOSOWANIE

Info

Publication number
PL2320952T3
PL2320952T3 PL09802455.7T PL09802455T PL2320952T3 PL 2320952 T3 PL2320952 T3 PL 2320952T3 PL 09802455 T PL09802455 T PL 09802455T PL 2320952 T3 PL2320952 T3 PL 2320952T3
Authority
PL
Poland
Prior art keywords
rtm
molecules
improved pre
splicing molecule
mrna trans
Prior art date
Application number
PL09802455.7T
Other languages
English (en)
Inventor
Lloyd G. Mitchell
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 filed Critical
Publication of PL2320952T3 publication Critical patent/PL2320952T3/pl

Links

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    • 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
    • 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
    • A61K48/005Medicinal 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P17/00Drugs for dermatological disorders
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P17/00Drugs for dermatological disorders
    • A61P17/02Drugs for dermatological disorders for treating wounds, ulcers, burns, scars, keloids, or the like
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P17/00Drugs for dermatological disorders
    • A61P17/06Antipsoriatics
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00Antineoplastic agents
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00Drugs for immunological or allergic disorders
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00Drugs for immunological or allergic disorders
    • A61P37/02Immunomodulators
    • A61P37/06Immunosuppressants, e.g. drugs for graft rejection
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/78Connective tissue peptides, e.g. collagen, elastin, laminin, fibronectin, vitronectin or cold insoluble globulin [CIG]
    • 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
    • C12N2740/00Reverse transcribing RNA viruses
    • C12N2740/00011Details
    • C12N2740/10011Retroviridae
    • C12N2740/13011Gammaretrovirus, e.g. murine leukeamia virus
    • C12N2740/13041Use of virus, viral particle or viral elements as a vector
    • C12N2740/13043Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
    • 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
    • C12N2840/00Vectors comprising a special translation-regulating system
    • C12N2840/44Vectors comprising a special translation-regulating system being a specific part of the splice mechanism, e.g. donor, acceptor
    • C12N2840/445Vectors comprising a special translation-regulating system being a specific part of the splice mechanism, e.g. donor, acceptor for trans-splicing, e.g. polypyrimidine tract, branch point splicing

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Molecular Biology (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Engineering & Computer Science (AREA)
  • Biophysics (AREA)
  • Wood Science & Technology (AREA)
  • Immunology (AREA)
  • Biomedical Technology (AREA)
  • Biochemistry (AREA)
  • Dermatology (AREA)
  • Physics & Mathematics (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Epidemiology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Toxicology (AREA)
  • Transplantation (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
PL09802455.7T 2008-07-30 2009-07-30 ULEPSZONE CZĄSTECZKI CZĄSTECZKI TRANS-SPLICINGUJĄCEJ PRE-mRNA (RTM) I ICH ZASTOSOWANIE PL2320952T3 (pl)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
EP20080013671 EP2151248A1 (en) 2008-07-30 2008-07-30 Improved pre-mRNA trans-splicing molecule (RTM) molecules and their uses

Publications (1)

Publication Number Publication Date
PL2320952T3 true PL2320952T3 (pl) 2016-11-30

Family

ID=39797958

Family Applications (1)

Application Number Title Priority Date Filing Date
PL09802455.7T PL2320952T3 (pl) 2008-07-30 2009-07-30 ULEPSZONE CZĄSTECZKI CZĄSTECZKI TRANS-SPLICINGUJĄCEJ PRE-mRNA (RTM) I ICH ZASTOSOWANIE

Country Status (6)

Country Link
US (1) US8735366B2 (pl)
EP (2) EP2151248A1 (pl)
JP (1) JP5735912B2 (pl)
ES (1) ES2579477T3 (pl)
PL (1) PL2320952T3 (pl)
WO (1) WO2010012472A1 (pl)

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CN102906535B (zh) * 2011-05-02 2016-01-20 福斯分析股份公司 光谱仪
WO2013025461A1 (en) * 2011-08-12 2013-02-21 Virxsys Corporation Compositions and methods for inducing apoptosis
HUE029977T2 (en) 2012-07-12 2017-04-28 Proqr Therapeutics Ii Bv Oligonucleotides to generate a change in the sequence of target RNA molecules present in a living cell
WO2014011050A1 (en) * 2012-07-12 2014-01-16 Proqr Therapeutics B.V. Exon replacement with stabilized artificial rnas
GB201219762D0 (en) 2012-11-02 2012-12-19 Bauer Johann A RNA trans-splicing molecule (RTM) for use in the treatment of cancer
ES2787198T3 (es) 2014-01-31 2020-10-15 Factor Bioscience Inc ARN sintético para su uso en el tratamiento de la epidermólisis ampollosa distrófica
GB201410693D0 (en) 2014-06-16 2014-07-30 Univ Southampton Splicing modulation
AU2015327836B2 (en) 2014-10-03 2021-07-01 Cold Spring Harbor Laboratory Targeted augmentation of nuclear gene output
WO2016131052A1 (en) 2015-02-13 2016-08-18 Factor Bioscience Inc. Nucleic acid products and methods of administration thereof
GB201504124D0 (en) 2015-03-11 2015-04-22 Proqr Therapeutics B V Oligonucleotides
GB201513151D0 (en) * 2015-07-27 2015-09-09 Univ Singapore Functional gene replacement therapy
KR102422625B1 (ko) 2015-10-09 2022-07-20 유니버시티 오브 사우스앰톤 유전자 발현의 조절 및 탈조절된 단백질 발현의 스크리닝
EP3377116A4 (en) 2015-11-19 2019-07-10 The Trustees of The University of Pennsylvania COMPOSITIONS AND METHODS FOR CORRECTING ECG-RELATED EYE DISEASE
US11096956B2 (en) 2015-12-14 2021-08-24 Stoke Therapeutics, Inc. Antisense oligomers and uses thereof
ES2882500T3 (es) 2015-12-14 2021-12-02 Cold Spring Harbor Laboratory Oligómeros antisentido para el tratamiento del síndrome de Dravet
KR101926331B1 (ko) * 2016-04-12 2018-12-07 (주)안트로젠 수포성 표피박리증 완화 또는 개선용 중간엽줄기세포-하이드로겔-생분해성 또는 중간엽줄기세포-하이드로겔-비분해성 지지체 조성물
EP3481432A4 (en) 2016-07-05 2020-05-06 The U.S.A. as represented by the Secretary, Department of Health and Human Services Diagnosing col6-related disorders and methods for treating same
CA3033788A1 (en) 2016-08-17 2018-02-22 Factor Bioscience Inc. Nucleic acid products and methods of administration thereof
IT201700094210A1 (it) * 2017-08-17 2019-02-17 Holostem Terapie Avanzate S R L Metodo per produrre lembi cellulari
AU2018322319B2 (en) 2017-08-25 2021-08-05 Stoke Therapeutics, Inc. Antisense oligomers for treatment of conditions and diseases
IT201700104587A1 (it) * 2017-09-19 2019-03-19 Holostem Terapie Avanzate S R L Usi terapeutici di lembi di cellule geneticamente modificate
US11993776B2 (en) 2018-04-17 2024-05-28 Ascidian Therapeutics, Inc. Trans-splicing molecules
CA3099280A1 (en) 2018-05-04 2019-11-07 Stoke Therapeutics, Inc. Methods and compositions for treatment of cholesteryl ester storage disease
US10501404B1 (en) 2019-07-30 2019-12-10 Factor Bioscience Inc. Cationic lipids and transfection methods
CA3150747A1 (en) * 2019-08-16 2021-02-25 Massachusetts Institute Of Technology Targeted trans-splicing using crispr/cas13
IL298063A (en) 2020-05-11 2023-01-01 Stoke Therapeutics Inc Opa1 antisense oligomers for treatment of conditions and diseases
CA3230419A1 (en) 2021-09-03 2023-03-09 David A. Nelles Rna editing via recruitment of spliceosome components
WO2024068898A1 (en) 2022-09-30 2024-04-04 Centre National De La Recherche Scientifique Therapy by trans-splicing of opa1 pre-messenger rnas for the treatment of diseases associated with opa1 gene mutations
WO2024118946A1 (en) * 2022-11-30 2024-06-06 Amber Bio Inc. Methods and compositions for targeted trans-splicing
WO2024206891A1 (en) * 2023-03-31 2024-10-03 Duke University Compositions for and methods of engineering the transcriptome

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US20060088938A1 (en) 1995-12-15 2006-04-27 Mitchell Lloyd G Methods and compositions for use in spliceosome mediated RNA trans-splicing in plants
CA2240494C (en) 1995-12-15 2007-03-13 Lloyd G. Mitchell Therapeutic molecules generated by trans-splicing
US6083702A (en) 1995-12-15 2000-07-04 Intronn Holdings Llc Methods and compositions for use in spliceosome mediated RNA trans-splicing
US6280978B1 (en) * 1995-12-15 2001-08-28 Intronn Holdings, Llc Methods and compositions for use in spliceosome mediated RNA trans-splicing
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Also Published As

Publication number Publication date
ES2579477T3 (es) 2016-08-11
EP2151248A1 (en) 2010-02-10
EP2320952B1 (en) 2016-05-04
US20130059901A1 (en) 2013-03-07
JP5735912B2 (ja) 2015-06-17
JP2011529333A (ja) 2011-12-08
EP2320952A1 (en) 2011-05-18
WO2010012472A1 (en) 2010-02-04
US8735366B2 (en) 2014-05-27

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