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PL3041958T3 - Redukcja rozpadu mRNA mediowanego sekwencjami nonsensownymi - Google Patents

Redukcja rozpadu mRNA mediowanego sekwencjami nonsensownymi

Info

Publication number
PL3041958T3
PL3041958T3 PL14842672T PL14842672T PL3041958T3 PL 3041958 T3 PL3041958 T3 PL 3041958T3 PL 14842672 T PL14842672 T PL 14842672T PL 14842672 T PL14842672 T PL 14842672T PL 3041958 T3 PL3041958 T3 PL 3041958T3
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
PL3041958T4 (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 PL3041958T4 publication Critical patent/PL3041958T4/pl
Publication of PL3041958T3 publication Critical patent/PL3041958T3/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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    • C12N15/00Mutation 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
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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
    • C12N2310/10Type of nucleic acid
    • C12N2310/11Antisense
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
    • C12N2310/315Phosphorothioates
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    • C12N2320/00Applications; Uses
    • C12N2320/10Applications; Uses in screening processes
    • C12N2320/11Applications; Uses in screening processes for the determination of target sites, i.e. of active nucleic acids
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    • C12N2320/31Combination therapy
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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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  • Pharmacology & Pharmacy (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Epidemiology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Analytical Chemistry (AREA)
  • Plant Pathology (AREA)
  • Dermatology (AREA)
  • Pathology (AREA)
  • Immunology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Neurology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Neurosurgery (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
PL14842672T 2013-09-04 2014-09-04 Redukcja rozpadu mRNA mediowanego sekwencjami nonsensownymi PL3041958T3 (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
PL3041958T4 PL3041958T4 (pl) 2020-11-02
PL3041958T3 true PL3041958T3 (pl) 2020-11-02

Family

ID=52628936

Family Applications (1)

Application Number Title Priority Date Filing Date
PL14842672T PL3041958T3 (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) PL3041958T3 (pl)
WO (1) WO2015035091A1 (pl)

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CA2930859C (en) 2013-09-04 2022-05-03 Cold Spring Harbor Laboratory Reducing nonsense-mediated mrna decay
GB201410693D0 (en) 2014-06-16 2014-07-30 Univ Southampton Splicing modulation
EP3201339A4 (en) 2014-10-03 2018-09-19 Cold Spring Harbor Laboratory Targeted augmentation of nuclear gene output
EP3359685A1 (en) 2015-10-09 2018-08-15 University Of Southampton Modulation of gene expression and screening for deregulated protein expression
CA3005131A1 (en) * 2015-12-14 2017-06-22 Cold Spring Harbor Laboratory Antisense oligomers for treatment of tuberous sclerosis complex
WO2017106211A1 (en) * 2015-12-14 2017-06-22 Cold Spring Harbor Laboratory Antisense oligomers for treatment of polycystic kidney disease
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
JP7054675B2 (ja) 2015-12-14 2022-04-14 コールド スプリング ハーバー ラボラトリー 網膜色素変性症18と網膜色素変性症13の処置のための組成物と方法
EP3390666A4 (en) * 2015-12-14 2019-08-07 Cold Spring Harbor Laboratory COMPOSITIONS AND METHODS OF TREATING NURSE DISEASES
WO2017106210A1 (en) 2015-12-14 2017-06-22 Cold Spring Harbor Laboratory Antisense oligomers for treatment of alagille syndrome
KR102604132B1 (ko) 2015-12-14 2023-11-17 콜드스프링하버러보러토리 상염색체 우성 정신 지체 5 및 드라베 증후군의 치료를 위한 안티센스 올리고머
EP3390634A4 (en) * 2015-12-14 2019-08-14 Cold Spring Harbor Laboratory COMPOSITIONS AND METHODS FOR THE TREATMENT OF EYE DISEASES
US11096956B2 (en) 2015-12-14 2021-08-24 Stoke Therapeutics, Inc. Antisense oligomers and uses thereof
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
JP7304697B2 (ja) 2015-12-23 2023-07-07 ムーンショット ファーマ エルエルシー 未成熟終止コドンのリードスルーを促進することにより免疫反応を誘発するための方法
EP3485041A1 (en) * 2016-07-14 2019-05-22 Universidade Do Porto Biomarker for prenatal diagnosis of twin-to-twin transfusion syndrome
EP3512516A1 (en) 2016-09-13 2019-07-24 Marco Cipolli Method of treatment of shwachman-diamond syndrome
FR3057774B1 (fr) * 2016-10-21 2025-11-07 Museum Nat Dhistoire Naturelle Derives de la purine pour leur utilisation dans le traitement ou la prevention de maladies dues a une mutation non-sens
WO2018237326A1 (en) 2017-06-22 2018-12-27 1AlMOONSHOT PHARMA LLC Methods for treating cancer with compositions comprising amlexanox and immune modulators
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WO2019053667A1 (en) * 2017-09-18 2019-03-21 Friedrich Miescher Institute For Biomedical Research INHIBITION OF AUTISTIC SPECTRUM DISORDER USING RIBOSOMIC TRANSLECTURE COMPOUNDS
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BR112020022512A2 (pt) 2018-05-04 2021-05-04 Stoke Therapeutics, Inc. métodos e composições para tratamento de doença de armazenamento de éster de colesteril
MX2021006146A (es) * 2018-11-29 2021-09-28 Eloxx Pharmaceuticals Metodos, composiciones y kits para tratar enfermedades oculares.
MX2021012428A (es) * 2019-04-10 2022-01-19 Ptc Therapeutics Inc Metodo para el tratamiento de distrofia muscular de duchenne mediada por mutacion sin sentido en pacientes pediatricos.
CN114025848A (zh) * 2019-04-24 2022-02-08 斯托克制药公司 用于调节剪接和翻译的方法和组合物
CN113785061B (zh) 2019-05-05 2025-04-29 耶路撒冷希伯来大学伊森姆研究发展有限公司 通过剪接调节恢复cftr功能
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Also Published As

Publication number Publication date
AU2014315183B2 (en) 2021-03-04
WO2015035091A1 (en) 2015-03-12
EP3041958A4 (en) 2017-04-26
EP3041958A1 (en) 2016-07-13
US20170159049A9 (en) 2017-06-08
PL3041958T4 (pl) 2020-11-02
CA2930859C (en) 2022-05-03
EP3041958B1 (en) 2019-12-04
US20160194630A1 (en) 2016-07-07
US11352624B2 (en) 2022-06-07
DK3041958T3 (da) 2020-03-09
ES2779302T3 (es) 2020-08-14
EP3690048A1 (en) 2020-08-05
US10647983B2 (en) 2020-05-12
AU2014315183A1 (en) 2016-04-21
US20200283762A1 (en) 2020-09-10
CA2930859A1 (en) 2015-03-12

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