CA3005247A1 - Oligomeres antisens pour le traitement de la polykystose renale - Google Patents
Oligomeres antisens pour le traitement de la polykystose renale Download PDFInfo
- Publication number
- CA3005247A1 CA3005247A1 CA3005247A CA3005247A CA3005247A1 CA 3005247 A1 CA3005247 A1 CA 3005247A1 CA 3005247 A CA3005247 A CA 3005247A CA 3005247 A CA3005247 A CA 3005247A CA 3005247 A1 CA3005247 A1 CA 3005247A1
- Authority
- CA
- Canada
- Prior art keywords
- nucleobases
- mrna
- fold
- retained intron
- protein
- Prior art date
- Legal status (The legal status 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 status listed.)
- Pending
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
- 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
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P13/00—Drugs for disorders of the urinary system
- A61P13/12—Drugs for disorders of the urinary system of the kidneys
-
- 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
- C12N15/111—General methods applicable to biologically active non-coding nucleic acids
-
- 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
- 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
-
- 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
- C12N2310/11—Antisense
-
- 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
- C12N2310/31—Chemical structure of the backbone
- C12N2310/315—Phosphorothioates
-
- 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
- C12N2310/32—Chemical structure of the sugar
- C12N2310/321—2'-O-R Modification
-
- 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
- C12N2320/33—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
-
- 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
- C12Q2600/156—Polymorphic or mutational markers
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Chemical & Material Sciences (AREA)
- Genetics & Genomics (AREA)
- Organic Chemistry (AREA)
- Biomedical Technology (AREA)
- Zoology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Wood Science & Technology (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Molecular Biology (AREA)
- General Health & Medical Sciences (AREA)
- Biochemistry (AREA)
- Microbiology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Plant Pathology (AREA)
- Analytical Chemistry (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Medicinal Chemistry (AREA)
- General Chemical & Material Sciences (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Pathology (AREA)
- 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)
Abstract
L'invention concerne des procédés et des compositions pour augmenter l'expression de PC-2, et pour traiter un sujet en ayant besoin, par exemple un sujet souffrant d'une déficience d'expression de protéine PC-2 ou un sujet atteint de polykystose rénale.
Applications Claiming Priority (3)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US201562267252P | 2015-12-14 | 2015-12-14 | |
US62/267,252 | 2015-12-14 | ||
PCT/US2016/066417 WO2017106211A1 (fr) | 2015-12-14 | 2016-12-13 | Oligomères antisens pour le traitement de la polykystose rénale |
Publications (1)
Publication Number | Publication Date |
---|---|
CA3005247A1 true CA3005247A1 (fr) | 2017-06-22 |
Family
ID=59057523
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
CA3005247A Pending CA3005247A1 (fr) | 2015-12-14 | 2016-12-13 | Oligomeres antisens pour le traitement de la polykystose renale |
Country Status (4)
Country | Link |
---|---|
EP (1) | EP3389782A4 (fr) |
JP (2) | JP2018538287A (fr) |
CA (1) | CA3005247A1 (fr) |
WO (1) | WO2017106211A1 (fr) |
Families Citing this family (8)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
GB201410693D0 (en) | 2014-06-16 | 2014-07-30 | Univ Southampton | Splicing modulation |
SG11201702682PA (en) | 2014-10-03 | 2017-04-27 | Cold Spring Harbor Lab | Targeted augmentation of nuclear gene output |
CA3000971A1 (fr) | 2015-10-09 | 2017-04-13 | University Of Southampton | Modulation de l'expression genique et criblage de l'expression de proteines deregulee |
US11096956B2 (en) | 2015-12-14 | 2021-08-24 | Stoke Therapeutics, Inc. | Antisense oligomers and uses thereof |
JP7049248B2 (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 | 常染色体優性精神遅滞-5とドラベ症候群の処置のためのアンチセンスオリゴマー |
AU2018322319B2 (en) | 2017-08-25 | 2021-08-05 | Stoke Therapeutics, Inc. | Antisense oligomers for treatment of conditions and diseases |
JP2022544702A (ja) * | 2019-08-19 | 2022-10-20 | ストーク セラピューティクス,インク. | スプライシングおよびタンパク質発現を調節するための組成物および方法 |
US20230287410A1 (en) | 2020-05-11 | 2023-09-14 | Stoke Therapeutics, Inc. | Opa1 antisense oligomers for treatment of conditions and diseases |
Family Cites Families (6)
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 |
CA2993381C (fr) * | 2006-07-24 | 2020-07-14 | Athena Diagnostics, Inc. | Procede de detection de l'apparition de la maladie polykystique renale autosomale dominante (mprad) en fonction de la presence d'une modification de sequence nucleotidique dans lapolykystose renale type dominant (pkd) 1 |
US20150141320A1 (en) * | 2012-05-16 | 2015-05-21 | Rana Therapeutics, Inc. | Compositions and methods for modulating gene expression |
PL3041958T3 (pl) * | 2013-09-04 | 2020-11-02 | Cold Spring Harbor Laboratory | Redukcja rozpadu mRNA mediowanego sekwencjami nonsensownymi |
SG11201702682PA (en) * | 2014-10-03 | 2017-04-27 | Cold Spring Harbor Lab | Targeted augmentation of nuclear gene output |
-
2016
- 2016-12-13 WO PCT/US2016/066417 patent/WO2017106211A1/fr active Application Filing
- 2016-12-13 EP EP16876500.6A patent/EP3389782A4/fr active Pending
- 2016-12-13 JP JP2018529219A patent/JP2018538287A/ja not_active Withdrawn
- 2016-12-13 CA CA3005247A patent/CA3005247A1/fr active Pending
-
2022
- 2022-01-04 JP JP2022000102A patent/JP2022046723A/ja active Pending
Also Published As
Publication number | Publication date |
---|---|
EP3389782A1 (fr) | 2018-10-24 |
JP2018538287A (ja) | 2018-12-27 |
EP3389782A4 (fr) | 2019-07-31 |
WO2017106211A1 (fr) | 2017-06-22 |
JP2022046723A (ja) | 2022-03-23 |
Similar Documents
Publication | Publication Date | Title |
---|---|---|
EP3390636B1 (fr) | Oligomères antisens destinés au traitement du syndrome de dravet | |
EP3390642B1 (fr) | Compositions et procédés pour le traitement de rétinite pigmentaire 13 | |
AU2015327836B2 (en) | Targeted augmentation of nuclear gene output | |
CA3005249A1 (fr) | Compositions et methodes de traitement de maladies renales | |
CA3005247A1 (fr) | Oligomeres antisens pour le traitement de la polykystose renale | |
CA3005090A1 (fr) | Compositions et procedes de traitement de maladies hepatiques | |
WO2017106210A1 (fr) | Oligomères antisens pour le traitement du syndrome d'alagille | |
WO2017106375A1 (fr) | Oligomères antisens pour le traitement de la sclérose tubéreuse de bourneville | |
CA3131591A1 (fr) | Oligomeres antisens pour le traitement d'etats pathologiques et autres maladies | |
US20240117353A1 (en) | Compositions for treatment of conditions and diseases associated with polycystin expression | |
WO2023235509A2 (fr) | Oligomères antisens pour le traitement de pathologies et de maladies fondées sur la dégradation des arn non-sens | |
WO2022271699A2 (fr) | Oligomères antisens pour le traitement d'états et de maladies fondées sur la dégradation des arnm non-sens | |
WO2024097138A1 (fr) | Oligomères antisens pour le traitement de pathologies et de maladies fondées sur la dégradation des arn à médiation non-sens |
Legal Events
Date | Code | Title | Description |
---|---|---|---|
EEER | Examination request |
Effective date: 20211213 |
|
EEER | Examination request |
Effective date: 20211213 |
|
EEER | Examination request |
Effective date: 20211213 |
|
EEER | Examination request |
Effective date: 20211213 |