MX2016004452A - Antisense oligonucleotide directed removal of proteolytic cleavage sites, the hchwa-d mutation, and trinucleotide repeat expansions. - Google Patents

Antisense oligonucleotide directed removal of proteolytic cleavage sites, the hchwa-d mutation, and trinucleotide repeat expansions.

Info

Publication number
MX2016004452A
MX2016004452A MX2016004452A MX2016004452A MX2016004452A MX 2016004452 A MX2016004452 A MX 2016004452A MX 2016004452 A MX2016004452 A MX 2016004452A MX 2016004452 A MX2016004452 A MX 2016004452A MX 2016004452 A MX2016004452 A MX 2016004452A
Authority
MX
Mexico
Prior art keywords
hchwa
mutation
proteolytic cleavage
trinucleotide repeat
antisense oligonucleotide
Prior art date
Application number
MX2016004452A
Other languages
Spanish (es)
Inventor
Maria Clasina VAN ROON-MOM Wilhelmina
Maurice EVERS Melvin
Antonius PEPERS Barry
Aartsma-Rus Annemieke
Jan Boudewijn Van Ommen Garrit-
Original Assignee
Academisch Ziekenhuis Leiden
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
Priority claimed from US14/047,876 external-priority patent/US20140039037A1/en
Application filed by Academisch Ziekenhuis Leiden filed Critical Academisch Ziekenhuis Leiden
Publication of MX2016004452A publication Critical patent/MX2016004452A/en

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
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • 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
    • C12Q1/6806—Preparing nucleic acids for analysis, e.g. for polymerase chain reaction [PCR] assay
    • 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
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Genetics & Genomics (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Neurology (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Biochemistry (AREA)
  • Analytical Chemistry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Neurosurgery (AREA)
  • Hospice & Palliative Care (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Immunology (AREA)
  • Psychiatry (AREA)
  • General Chemical & Material Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Plant Pathology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)

Abstract

The invention relates to means and methods for removing a proteolytic cleavage site, the HCHWA-D mutation or the amino acids encoded by a trinucleotide repeat expansion from a protein comprising providing a cell that expresses pre-m RNA encoding said protein with an anti-sense oligonucleotide that induces skipping of the exonic sequence that comprises said proteolytic cleavage site, HCHWA-D mutation or trinucleotide repeat expansion, respectively, the method further comprising allowing translation of m RNA produced from said pre-m RNA.
MX2016004452A 2013-10-07 2014-10-07 Antisense oligonucleotide directed removal of proteolytic cleavage sites, the hchwa-d mutation, and trinucleotide repeat expansions. MX2016004452A (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US14/047,876 US20140039037A1 (en) 2010-08-05 2013-10-07 Antisense oligonucleotide directed removal of proteolytic cleavage sites, the hchwa-d mutation, and trinucleotide repeat expansions
PCT/NL2014/050698 WO2015053624A2 (en) 2013-10-07 2014-10-07 Antisense oligonucleotide directed removal of proteolytic cleavage sites, the hchwa-d mutation, and trinucleotide repeat expansions

Publications (1)

Publication Number Publication Date
MX2016004452A true MX2016004452A (en) 2017-04-27

Family

ID=51844811

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2016004452A MX2016004452A (en) 2013-10-07 2014-10-07 Antisense oligonucleotide directed removal of proteolytic cleavage sites, the hchwa-d mutation, and trinucleotide repeat expansions.

Country Status (3)

Country Link
MX (1) MX2016004452A (en)
RU (1) RU2692634C2 (en)
WO (1) WO2015053624A2 (en)

Families Citing this family (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3353303B1 (en) 2015-09-25 2023-08-02 Ionis Pharmaceuticals, Inc. Compositions and methods for modulating ataxin 3 expression
AU2016337034A1 (en) * 2015-10-16 2018-05-10 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for use in treating Alzheimer's disease
JOP20190104A1 (en) 2016-11-10 2019-05-07 Ionis Pharmaceuticals Inc Compounds and methods for reducing atxn3 expression
GB201714027D0 (en) * 2017-09-01 2017-10-18 Proqr Therapeutics Ii Bv Antisense oligonucleotides for the treatment of huntington's disease
US11732260B2 (en) 2018-03-02 2023-08-22 Ionis Pharmaceuticals, Inc. Compounds and methods for the modulation of amyloid-β precursor protein
MX2020011911A (en) 2018-05-09 2021-01-29 Ionis Pharmaceuticals Inc Compounds and methods for reducing atxn3 expression.
CN115667513A (en) 2020-05-12 2023-01-31 田边三菱制药株式会社 Compounds, methods and pharmaceutical compositions for modulating Ataxin 3 expression

Family Cites Families (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
DE69229718D1 (en) * 1992-03-20 1999-09-09 Innogenetics Nv Mutated form of the beta amyloid precursor protein gene
UY29504A1 (en) * 2005-04-29 2006-10-31 Rinat Neuroscience Corp DIRECTED ANTIBODIES AGAINST BETA AMYLOID PEPTIDE AND METHODS USING THE SAME.
CA2660523C (en) * 2006-08-11 2019-03-19 Prosensa Technologies B.V. Methods and means for treating dna repeat instability associated genetic disorders
DK2601294T3 (en) * 2010-08-05 2019-02-18 Academisch Ziekenhuis Leiden ANTISENSE OLIGONUCLEOTIDE CALCULATED FOR THE REMOVAL OF PROTEOLYTIC DIVISION PLACES FROM PROTEINS

Also Published As

Publication number Publication date
RU2016114321A (en) 2017-11-15
RU2016114321A3 (en) 2018-08-09
RU2692634C2 (en) 2019-06-25
WO2015053624A2 (en) 2015-04-16
WO2015053624A3 (en) 2015-08-27

Similar Documents

Publication Publication Date Title
MX2016004452A (en) Antisense oligonucleotide directed removal of proteolytic cleavage sites, the hchwa-d mutation, and trinucleotide repeat expansions.
CL2016002316A1 (en) Method to cause a genetic change in a plant cell, based on subjecting said cell to a DNA cut and a modified gron.
NZ607297A (en) Antisense oligonucleotide directed removal of proteolytic cleavage sites from proteins
AR122864A2 (en) LIPASE VARIANTS AND POLYNUCLEOTIDES ENCODING THEM
BRPI0917353A2 (en) method and system for optimized production of gnl.
UA116980C2 (en) Glyphosate resistant plants and associated methods
BRPI1011160A2 (en) phytases, nucleic acids encoding them and methods for producing and using them
EP2857694A4 (en) Erosion prediction method, erosion prediction system, erosion characteristics database used in this prediction, and method for constructing same
EP2692888A4 (en) Case hardening steel, method for producing same, and mechanical structural part using case hardening steel
ES2721309T3 (en) Composition for preparing tagatose and method for preparing tagatose from fructose
WO2012156615A3 (en) Power supply system and method for eliminating the pogo effect
BRPI1008004A2 (en) method for metal production and apparatus.
UA111979C2 (en) METHOD OF LUTEINE EXTRACTION FROM NATURAL MATERIALS
UA105785C2 (en) Process for purifying lipopeptides
MY181068A (en) Recombinant microorganism for improved production of fine chemicals
MX2013009855A (en) Plants having enhanced yield-related traits and producing methods thereof.
EP2684141A4 (en) Apparatus, system and method for a vector-based form field document
MY183321A (en) Microorganism producing o-acetyl-homoserine and method for producing o-acetylhomoserine using the same
PL2562272T3 (en) Method for producing steel product or steel component having excellent mechanical properties, steel product produced by the method and use of steel pipe made of strain hardened steel
MX342151B (en) Sucker rod apparatus and method.
PH12014500550A1 (en) Plants having enhanced yield-related traits and a method for making the same
EP3603849A4 (en) Method for producing austenite stainless steel slab
MX356270B (en) Methods for decontaminating circuits for producing glucose polymers and hydrolysates of glucose polymers.
WO2012141895A3 (en) Rare earth removal of phosphorus-containing materials
IL227592A0 (en) Method for producing 2,2-difluoroethylamine starting from 2,2-difluoro-1-chloroethane