PL3041958T4 - Redukcja rozpadu mRNA mediowanego sekwencjami nonsensownymi - Google Patents

Redukcja rozpadu mRNA mediowanego sekwencjami nonsensownymi

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Publication number
PL3041958T4
PL3041958T4 PL14842672T PL14842672T PL3041958T4 PL 3041958 T4 PL3041958 T4 PL 3041958T4 PL 14842672 T PL14842672 T PL 14842672T PL 14842672 T PL14842672 T PL 14842672T PL 3041958 T4 PL3041958 T4 PL 3041958T4
Authority
PL
Poland
Prior art keywords
mrna decay
mediated mrna
nonsense
reducing
reducing nonsense
Prior art date
Application number
PL14842672T
Other languages
English (en)
Other versions
PL3041958T3 (pl
Inventor
Adrian Krainer
Isabel AZNAREZ
Tomoki NOMAKUCHI
Original Assignee
Cold Spring Harbor Laboratory
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 Cold Spring Harbor Laboratory filed Critical Cold Spring Harbor Laboratory
Publication of PL3041958T3 publication Critical patent/PL3041958T3/pl
Publication of PL3041958T4 publication Critical patent/PL3041958T4/pl

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    • 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
    • C12N15/111General methods applicable to biologically active non-coding nucleic acids
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/7105Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/33Heterocyclic compounds
    • A61K31/395Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/41Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having five-membered rings with two or more ring hetero atoms, at least one of which being nitrogen, e.g. tetrazole
    • A61K31/4245Oxadiazoles
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/713Double-stranded nucleic acids or oligonucleotides
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/715Polysaccharides, i.e. having more than five saccharide radicals attached to each other by glycosidic linkages; Derivatives thereof, e.g. ethers, esters
    • A61K31/726Glycosaminoglycans, i.e. mucopolysaccharides
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
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    • 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
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    • C12Q1/00Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
    • C12Q1/68Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving nucleic acids
    • C12Q1/6876Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes
    • C12Q1/6883Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes for diseases caused by alterations of genetic material
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    • C12N2310/00Structure or type of the nucleic acid
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
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    • C12Q2600/00Oligonucleotides characterized by their use
    • C12Q2600/106Pharmacogenomics, i.e. genetic variability in individual responses to drugs and drug metabolism
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    • C12Q2600/00Oligonucleotides characterized by their use
    • C12Q2600/156Polymorphic or mutational markers

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  • Proteomics, Peptides & Aminoacids (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
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  • Neurosurgery (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
PL14842672T 2013-09-04 2014-09-04 Redukcja rozpadu mRNA mediowanego sekwencjami nonsensownymi PL3041958T4 (pl)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201361873780P 2013-09-04 2013-09-04
PCT/US2014/054151 WO2015035091A1 (en) 2013-09-04 2014-09-04 Reducing nonsense-mediated mrna decay
EP14842672.9A EP3041958B1 (en) 2013-09-04 2014-09-04 Reducing nonsense-mediated mrna decay

Publications (2)

Publication Number Publication Date
PL3041958T3 PL3041958T3 (pl) 2020-11-02
PL3041958T4 true PL3041958T4 (pl) 2020-11-02

Family

ID=52628936

Family Applications (1)

Application Number Title Priority Date Filing Date
PL14842672T PL3041958T4 (pl) 2013-09-04 2014-09-04 Redukcja rozpadu mRNA mediowanego sekwencjami nonsensownymi

Country Status (8)

Country Link
US (2) US10647983B2 (pl)
EP (2) EP3041958B1 (pl)
AU (1) AU2014315183B2 (pl)
CA (1) CA2930859C (pl)
DK (1) DK3041958T3 (pl)
ES (1) ES2779302T3 (pl)
PL (1) PL3041958T4 (pl)
WO (1) WO2015035091A1 (pl)

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EP3041958B1 (en) 2013-09-04 2019-12-04 Cold Spring Harbor Laboratory Reducing nonsense-mediated mrna decay
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
CN108603230A (zh) * 2015-10-09 2018-09-28 南安普敦大学 基因表达的调节与蛋白质表达失调的筛选
EP3390666A4 (en) * 2015-12-14 2019-08-07 Cold Spring Harbor Laboratory COMPOSITIONS AND METHODS OF TREATING NURSE DISEASES
EP3390635A4 (en) * 2015-12-14 2019-05-01 Cold Spring Harbor Laboratory Antisense oligomers for the treatment of a tuberous sclerosis complex
WO2017106377A1 (en) * 2015-12-14 2017-06-22 Cold Spring Harbor Laboratory Antisense oligomers for treatment of autosomal dominant mental retardation-5 and dravet syndrome
JP7049249B2 (ja) 2015-12-14 2022-04-06 コールド スプリング ハーバー ラボラトリー 中枢神経系疾患の処置のための組成物および方法
US11096956B2 (en) 2015-12-14 2021-08-24 Stoke Therapeutics, Inc. Antisense oligomers and uses thereof
CA3005245A1 (en) 2015-12-14 2017-06-22 Cold Spring Harbor Laboratory Antisense oligomers for treatment of alagille syndrome
JP2018538287A (ja) * 2015-12-14 2018-12-27 コールド スプリング ハーバー ラボラトリー 多発性嚢胞腎の処置のためのアンチセンスオリゴマー
JP7036723B2 (ja) * 2015-12-14 2022-03-15 コールド スプリング ハーバー ラボラトリー 眼疾患の処置のための組成物および方法
JP2019500345A (ja) 2015-12-14 2019-01-10 コールド スプリング ハーバー ラボラトリー 肝臓病の処置のための組成物および方法
ES2903394T3 (es) 2015-12-14 2022-04-01 Cold Spring Harbor Laboratory Composiciones para el tratamiento de la retinitis pigmentosa 13
US11135221B2 (en) 2015-12-23 2021-10-05 Moonshot Pharma Llc Methods for inducing an immune response
AU2017294696A1 (en) * 2016-07-14 2019-01-03 Ipatimup ( Instituto De Patologia E Imunologia Molecular Da Universidade Do Porto) Biomarker for prenatal diagnosis of twin-to-twin transfusion syndrome
WO2018050706A1 (en) * 2016-09-13 2018-03-22 Marco Cipolli Method of treatment of shwachman-diamond syndrome
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JP2020525434A (ja) 2017-06-22 2020-08-27 ムーンショット ファーマ エルエルシー アンレキサノクス及び免疫調節剤を含む組成物で癌を治療する方法
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JP2022510996A (ja) * 2018-11-29 2022-01-28 エロックス・ファーマシューティカルズ 眼疾患を治療する方法、組成物及びキット
AU2020227825B2 (en) 2019-02-27 2026-03-26 Stoke Therapeutics, Inc. Antisense oligomers for treatment of conditions and diseases
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Also Published As

Publication number Publication date
AU2014315183B2 (en) 2021-03-04
CA2930859C (en) 2022-05-03
EP3690048A1 (en) 2020-08-05
WO2015035091A1 (en) 2015-03-12
US20160194630A1 (en) 2016-07-07
PL3041958T3 (pl) 2020-11-02
US11352624B2 (en) 2022-06-07
DK3041958T3 (da) 2020-03-09
EP3041958B1 (en) 2019-12-04
US10647983B2 (en) 2020-05-12
EP3041958A4 (en) 2017-04-26
US20200283762A1 (en) 2020-09-10
EP3041958A1 (en) 2016-07-13
CA2930859A1 (en) 2015-03-12
US20170159049A9 (en) 2017-06-08
AU2014315183A1 (en) 2016-04-21
ES2779302T3 (es) 2020-08-14

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