JP2018538287A - 多発性嚢胞腎の処置のためのアンチセンスオリゴマー - Google Patents

多発性嚢胞腎の処置のためのアンチセンスオリゴマー Download PDF

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JP2018538287A
JP2018538287A JP2018529219A JP2018529219A JP2018538287A JP 2018538287 A JP2018538287 A JP 2018538287A JP 2018529219 A JP2018529219 A JP 2018529219A JP 2018529219 A JP2018529219 A JP 2018529219A JP 2018538287 A JP2018538287 A JP 2018538287A
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protein
nucleobases
intron
mrna precursor
retained intron
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JP2018538287A5 (enExample
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アズナレズ，イザベル
エム． ナッシュ，ヒュー
エム． ナッシュ，ヒュー
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コールド スプリング ハーバー ラボラトリー
コールド スプリング ハーバー ラボラトリー
ストーク セラピューティクス，インク．
ストーク セラピューティクス，インク．
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Publication of JP2018538287A publication Critical patent/JP2018538287A/ja
Publication of JP2018538287A5 publication Critical patent/JP2018538287A5/ja
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    • 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
    • C12N15/1138—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 against receptors or cell surface proteins
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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
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    • C12Q1/00—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
    • C12Q1/68—Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving nucleic acids
    • C12Q1/6876—Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes
    • C12Q1/6883—Nucleic 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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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
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    • 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
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • 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
    • C12Q—MEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
    • C12Q2600/00—Oligonucleotides characterized by their use
    • C12Q2600/106—Pharmacogenomics, i.e. genetic variability in individual responses to drugs and drug metabolism
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    • C12Q—MEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
    • C12Q2600/00—Oligonucleotides characterized by their use
    • C12Q2600/156—Polymorphic or mutational markers

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  • Health & Medical Sciences (AREA)
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  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Biomedical Technology (AREA)
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  • Bioinformatics & Cheminformatics (AREA)
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  • Proteomics, Peptides & Aminoacids (AREA)
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  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
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  • Chemical Kinetics & Catalysis (AREA)
  • Urology & Nephrology (AREA)
  • Immunology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
JP2018529219A 2015-12-14 2016-12-13 多発性嚢胞腎の処置のためのアンチセンスオリゴマー Withdrawn JP2018538287A (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2022000102A JP2022046723A (ja) 2015-12-14 2022-01-04 多発性嚢胞腎の処置のためのアンチセンスオリゴマー

Applications Claiming Priority (3)

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US201562267252P 2015-12-14 2015-12-14
US62/267,252 2015-12-14
PCT/US2016/066417 WO2017106211A1 (en) 2015-12-14 2016-12-13 Antisense oligomers for treatment of polycystic kidney disease

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JP2022000102A Division JP2022046723A (ja) 2015-12-14 2022-01-04 多発性嚢胞腎の処置のためのアンチセンスオリゴマー

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JP2018538287A true JP2018538287A (ja) 2018-12-27
JP2018538287A5 JP2018538287A5 (enExample) 2020-01-30

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JP2022000102A Pending JP2022046723A (ja) 2015-12-14 2022-01-04 多発性嚢胞腎の処置のためのアンチセンスオリゴマー

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EP (1) EP3389782A4 (enExample)
JP (2) JP2018538287A (enExample)
CA (1) CA3005247A1 (enExample)
WO (1) WO2017106211A1 (enExample)

Families Citing this family (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
US11096956B2 (en) 2015-12-14 2021-08-24 Stoke Therapeutics, Inc. Antisense oligomers and uses thereof
KR102604132B1 (ko) 2015-12-14 2023-11-17 콜드스프링하버러보러토리 상염색체 우성 정신 지체 5 및 드라베 증후군의 치료를 위한 안티센스 올리고머
SI3673080T1 (sl) 2017-08-25 2024-03-29 Stoke Therapeutics, Inc. Protismiselni oligomeri za zdravljenje bolezenskih stanj in bolezni
BR112020022512A2 (pt) 2018-05-04 2021-05-04 Stoke Therapeutics, Inc. métodos e composições para tratamento de doença de armazenamento de éster de colesteril
CN114746550A (zh) * 2019-08-19 2022-07-12 斯托克制药公司 用于调节剪接和蛋白质表达的组合物和方法
AU2021270720A1 (en) 2020-05-11 2022-12-08 Stoke Therapeutics, Inc. OPA1 antisense oligomers for treatment of conditions and diseases
AU2022215577A1 (en) * 2021-02-03 2023-09-21 Stoke Therapeutics, Inc. Compositions for treatment of conditions and diseases associated with polycystin expression
WO2024254639A1 (en) * 2023-06-16 2024-12-19 PYC Therapeutics Limited Compositions and methods for treatment of kidney disease

Family Cites Families (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20040102402A1 (en) * 2002-11-22 2004-05-27 Isis Pharmaceuticals Inc. Modulation of tissue factor expression
US8007790B2 (en) * 2006-04-03 2011-08-30 Stowers Institute For Medical Research Methods for treating polycystic kidney disease (PKD) or other cyst forming diseases
WO2008094194A2 (en) * 2006-07-24 2008-08-07 Athena Diagnostics, Inc. Pkd mutations and evaluation of same
EP2850184A4 (en) * 2012-05-16 2016-01-27 Rana Therapeutics Inc COMPOSITIONS AND METHOD FOR MODULATING GENE EXPRESSION
CA2930859C (en) * 2013-09-04 2022-05-03 Cold Spring Harbor Laboratory Reducing nonsense-mediated mrna decay
EP3201339A4 (en) * 2014-10-03 2018-09-19 Cold Spring Harbor Laboratory Targeted augmentation of nuclear gene output

Non-Patent Citations (8)

* Cited by examiner, † Cited by third party
Title
CLIN. GENET., vol. 80, JPN6020042398, 2011, pages 287 - 292, ISSN: 0004381760 *
FLUID MANAGEMENT RENAISSANCE, vol. 4, no. 3, JPN6020042395, 2014, pages 280 - 285, ISSN: 0004381757 *
J. AM. SOC. NEPHROL., vol. 10, JPN6020042392, 1999, pages 2342 - 2351, ISSN: 0004381754 *
J. HUM. GENET., vol. 46, JPN6020042397, 2001, pages 511 - 517, ISSN: 0004381759 *
MOL. BIOL. REP., vol. 37, JPN6020042396, 2010, pages 2951 - 2958, ISSN: 0004381758 *
NUCLEIC ACIDS RESEARCH, vol. 42, no. 12, JPN6020042391, 2014, pages 8161 - 8173, ISSN: 0004381753 *
THE JOURNAL OF BIOLOGICAL CHEMISTRY, vol. 274, no. 51, JPN6020042394, 1999, pages 36193 - 36199, ISSN: 0004381756 *
THE JOURNAL OF CLINICAL INVESTIGATION, vol. 112, no. 4, JPN6020042393, 2003, pages 481 - 486, ISSN: 0004381755 *

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Publication number Publication date
EP3389782A1 (en) 2018-10-24
WO2017106211A1 (en) 2017-06-22
EP3389782A4 (en) 2019-07-31
CA3005247A1 (en) 2017-06-22
JP2022046723A (ja) 2022-03-23

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