PL405224A1 - Cząsteczka kwasu nukleinowego, kaseta ekspresyjna, wektor ekspresyjny, komórka eukariotycznego gospodarza, transgeniczny ssak, sposób indukowania interferencji RNA w eukariotycznym gospodarzu i zastosowanie cząsteczki kwasu nukleinowego w terapii chorób wywoływanych ekspansją trójnukleotydowych powtórzeń typu CAG - Google Patents

Cząsteczka kwasu nukleinowego, kaseta ekspresyjna, wektor ekspresyjny, komórka eukariotycznego gospodarza, transgeniczny ssak, sposób indukowania interferencji RNA w eukariotycznym gospodarzu i zastosowanie cząsteczki kwasu nukleinowego w terapii chorób wywoływanych ekspansją trójnukleotydowych powtórzeń typu CAG

Info

Publication number
PL405224A1
PL405224A1 PL405224A PL40522413A PL405224A1 PL 405224 A1 PL405224 A1 PL 405224A1 PL 405224 A PL405224 A PL 405224A PL 40522413 A PL40522413 A PL 40522413A PL 405224 A1 PL405224 A1 PL 405224A1
Authority
PL
Poland
Prior art keywords
nucleic acid
acid molecule
eukaryotic host
expansion
rna interference
Prior art date
Application number
PL405224A
Other languages
English (en)
Other versions
PL243776B1 (pl
Inventor
Włodzimierz Krzyżosiak
Marta Olejniczak
Paulina Gałka-Marciniak
Agnieszka Fiszer
Original Assignee
Instytut Chemii Bioorganicznej Polskiej Akademii Nauk
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 Instytut Chemii Bioorganicznej Polskiej Akademii Nauk filed Critical Instytut Chemii Bioorganicznej Polskiej Akademii Nauk
Priority to PL405224A priority Critical patent/PL243776B1/pl
Priority to EP14781962.7A priority patent/EP3041936A2/en
Priority to US14/916,039 priority patent/US9970004B2/en
Priority to PCT/PL2014/000100 priority patent/WO2015030616A2/en
Publication of PL405224A1 publication Critical patent/PL405224A1/pl
Priority to US15/954,561 priority patent/US10329566B2/en
Publication of PL243776B1 publication Critical patent/PL243776B1/pl

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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • 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
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • 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
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • C12N2310/141—MicroRNAs, miRNAs
    • 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
    • C12N2310/33—Chemical structure of the base
    • C12N2310/334—Modified C
    • 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
    • C12N2310/33—Chemical structure of the base
    • C12N2310/335—Modified T or U
    • 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
    • C12N2310/33—Chemical structure of the base
    • C12N2310/336—Modified G
    • 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
    • C12N2310/50—Physical structure
    • C12N2310/53—Physical structure partially self-complementary or closed
    • C12N2310/531—Stem-loop; Hairpin
    • 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
    • C12N2310/50—Physical structure
    • C12N2310/53—Physical structure partially self-complementary or closed
    • C12N2310/533—Physical structure partially self-complementary or closed having a mismatch or nick in at least one of the strands
    • 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
    • C12N2320/34—Allele or polymorphism specific uses
    • 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
    • C12N2330/00—Production
    • C12N2330/50—Biochemical production, i.e. in a transformed host cell
    • C12N2330/51—Specially adapted vectors

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Chemical & Material Sciences (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Molecular Biology (AREA)
  • Wood Science & Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • General Chemical & Material Sciences (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Psychology (AREA)
  • Medicinal Chemistry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)

Abstract

Przedmiotem wynalazku są: cząsteczka kwasu nukleinowego, kaseta ekspresyjna, wektor ekspresyjny, komórka eukariotycznego gospodarza, sposób indukowania interferencji RNA w eukariotycznym gospodarzu i zastosowanie cząsteczki kwasu nukleinowego w terapii chorób wywoływanych ekspansją trójnukleotydowych powtórzeń typu CAG. Rozwiązanie dotyczy nowej koncepcji leczenia dziedzicznych chorób neurologicznych u człowieka wywoływanych ekspansją powtórzeń trójnukleotydowych typu CAG, z wykorzystaniem technologii interferencji RNA.
PL405224A 2013-09-02 2013-09-02 Cząsteczka kwasu nukleinowego, kaseta ekspresyjna, wektor ekspresyjny, komórka eukariotycznego gospodarza i zastosowanie cząsteczki kwasu nukleinowego w terapii chorób wywoływanych ekspansją trójnukleotydowych powtórzeń typu CAG PL243776B1 (pl)

Priority Applications (5)

Application Number Priority Date Filing Date Title
PL405224A PL243776B1 (pl) 2013-09-02 2013-09-02 Cząsteczka kwasu nukleinowego, kaseta ekspresyjna, wektor ekspresyjny, komórka eukariotycznego gospodarza i zastosowanie cząsteczki kwasu nukleinowego w terapii chorób wywoływanych ekspansją trójnukleotydowych powtórzeń typu CAG
EP14781962.7A EP3041936A2 (en) 2013-09-02 2014-09-02 Nucleic acid molecule, expression cassette, expression vector, eukaryotic host cell, induction method of rna interference in eukaryotic host and use of nucleic acid molecule in therapy of diseases induced by expansion of trinucleotide cag repeats
US14/916,039 US9970004B2 (en) 2013-09-02 2014-09-02 Nucleic acid molecule, expression cassette, expression vector, eukaryotic host cell, induction method of RNA interferencde in eukaryotic host and use of the nucleic acid molecule in therapy of diseases induced by expansion of trinucleotide CAG repeats
PCT/PL2014/000100 WO2015030616A2 (en) 2013-09-02 2014-09-02 Nucleic acid molecule, expression cassette, expression vector, eukaryotic host cell, induction method of rna interference in eukaryotic host and use of nucleic acid molecule in therapy of diseases induced by expansion of trinucleotide cag repeats
US15/954,561 US10329566B2 (en) 2013-09-02 2018-04-16 Nucleic acid molecule, expression cassette, expression vector, eukaryotic host cell, induction method of RNA interference in eukaryotic host and use of the nucleic acid molecule in therapy of diseases induced by expansion of trinucleotide CAG repeats

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
PL405224A PL243776B1 (pl) 2013-09-02 2013-09-02 Cząsteczka kwasu nukleinowego, kaseta ekspresyjna, wektor ekspresyjny, komórka eukariotycznego gospodarza i zastosowanie cząsteczki kwasu nukleinowego w terapii chorób wywoływanych ekspansją trójnukleotydowych powtórzeń typu CAG

Publications (2)

Publication Number Publication Date
PL405224A1 true PL405224A1 (pl) 2015-03-16
PL243776B1 PL243776B1 (pl) 2023-10-09

Family

ID=51688382

Family Applications (1)

Application Number Title Priority Date Filing Date
PL405224A PL243776B1 (pl) 2013-09-02 2013-09-02 Cząsteczka kwasu nukleinowego, kaseta ekspresyjna, wektor ekspresyjny, komórka eukariotycznego gospodarza i zastosowanie cząsteczki kwasu nukleinowego w terapii chorób wywoływanych ekspansją trójnukleotydowych powtórzeń typu CAG

Country Status (4)

Country Link
US (2) US9970004B2 (pl)
EP (1) EP3041936A2 (pl)
PL (1) PL243776B1 (pl)
WO (1) WO2015030616A2 (pl)

Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US11535851B2 (en) * 2016-05-05 2022-12-27 Benitec IP Holdings Inc. Reagents for treatment of hepatitis B virus (HBV) infection and use thereof
WO2020104295A1 (en) * 2018-11-19 2020-05-28 Uniqure Ip B.V. Rnai induced reduction of ataxin-3 for the treatment of spinocerebellar ataxia type 3
WO2023060104A1 (en) * 2021-10-06 2023-04-13 Iris Medicine, Inc. Compositions and methods for treating cag repeat diseases
AU2024253981A1 (en) * 2023-04-05 2025-11-06 Board Of Trustees Of Southern Illinois University Compositions and methods for treating cag repeat diseases
US20240360443A1 (en) 2023-04-09 2024-10-31 Dystrogen Gene Therapies Inc. Nucleic acid molecule and use of the nucleic acid molecule in therapy of diseases induced by expansion of trinucleotide cag repeats

Family Cites Families (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2003013437A2 (en) 2001-08-07 2003-02-20 University Of Delaware Compositions and methods for the prevention and treatment of huntington's disease
KR20050026384A (ko) * 2002-04-26 2005-03-15 내셔날 인스티튜트 오브 어드밴스드 인더스트리얼 사이언스 앤드 테크놀로지 ＲＮＡｉ효과를 가지는 스템 루프 형 ＲＮＡ 분자 발현 시스템
US20080274989A1 (en) * 2002-08-05 2008-11-06 University Of Iowa Research Foundation Rna Interference Suppression of Neurodegenerative Diseases and Methods of Use Thereof
US20050255086A1 (en) * 2002-08-05 2005-11-17 Davidson Beverly L Nucleic acid silencing of Huntington's Disease gene
WO2011097388A1 (en) 2010-02-03 2011-08-11 Alnylam Pharmaceuticals, Inc. Selective inhibition of polyglutamine protein expression
US8993532B2 (en) * 2010-04-23 2015-03-31 Cold Spring Harbor Laboratory Structurally designed shRNAs
WO2012109667A1 (en) 2011-02-12 2012-08-16 University Of Iowa Research Foundation Therapeutic compounds
WO2013033223A1 (en) * 2011-08-29 2013-03-07 Isis Pharmaceuticals, Inc. Methods and compounds useful in conditions related to repeat expansion

Also Published As

Publication number Publication date
US20190024081A1 (en) 2019-01-24
WO2015030616A2 (en) 2015-03-05
WO2015030616A4 (en) 2015-10-01
US10329566B2 (en) 2019-06-25
PL243776B1 (pl) 2023-10-09
US9970004B2 (en) 2018-05-15
US20160376586A1 (en) 2016-12-29
EP3041936A2 (en) 2016-07-13
WO2015030616A3 (en) 2015-09-03

Similar Documents

Publication Publication Date Title
CY1124168T1 (el) Ετεροκυκλυλαμiνες ως αναστολεiς ρι3κ
CY1122707T1 (el) Ετεροκυκλικοι αναστολεις γλουταμινασης
CY1124407T1 (el) Ενωσεις αναστολεα αυτοταξινης
LTPA2020510I1 (lt) Pakeistieji 4-fenil-piridinai, skirti su NK-receptoriumi susijusių ligų gydymui
EP3579858A4 (en) GENE THERAPY AGAINST HAPLOINSUFFICIENCY
DK3706796T3 (da) Eksosomerer som RNA terapeutikske midler
IL261563A (en) Methods and compositions for rna-directed target dna modification and for rna-directed modulation of transcription
CY1120246T1 (el) Ενωσεις αναστολεων μεταλλοενζυμου
DK3289080T3 (da) Genterapi til autosomalt dominante sygdomme
PT3313991T (pt) Fator ix modificado e composições, métodos e utilizações para transferência de genes para células, órgãos e tecidos
DK2631291T3 (da) Nukleinsyremolekyler, der inducerer rna-interferens, og anvendelser deraf
IL284447A (en) Dux4 rna silencing using rna targeting crispr-cas13b
HUE045629T2 (hu) Készítmények, alkalmazások és eljárások anyagcsere rendellenességek és betegségek kezelésére
IL259876B (en) Augmented acid alpha-glucosidase for the treatment of pompe disease
EA202091568A1 (ru) Ингибиторы глюкозилцерамид-синтазы
DK2922828T3 (da) 4,6-diamino-pyrimidin-derivater som bmi-1-inhibitorer til at behandle cancer
HUE051632T2 (hu) Betegségek kezelésére szolgáló készítmények és eljárások
PL405224A1 (pl) Cząsteczka kwasu nukleinowego, kaseta ekspresyjna, wektor ekspresyjny, komórka eukariotycznego gospodarza, transgeniczny ssak, sposób indukowania interferencji RNA w eukariotycznym gospodarzu i zastosowanie cząsteczki kwasu nukleinowego w terapii chorób wywoływanych ekspansją trójnukleotydowych powtórzeń typu CAG
IL277695B1 (en) Pladienolide derivatives as spliceosome targeting agents for treating cancer
EP3750553A4 (en) THERAPEUTIC FOR PSORIASIS
CY1121859T1 (el) Μεθοδος παρασκευης αναστολεων συνθασης γλυκοζυλοκεραμιδης
BR112016001978A2 (pt) derivados de ciclopentano 1,3-dissubstituído
DK3652326T3 (da) Midler og fremgangsmåder til AAV-genterapi i mennesker
EP3568694A4 (en) DOXORUBICIN-GOLD NANOCONJUGATES TARGETED FOR ANTITUMOR THERAPY
EP2957289A4 (en) AMPLIFIER OF THE ACTIVITY OF AN OXIDIZED PROTEIN HYDROLASE