CO2017011617A2 - Agentes nucleósidos para la reducción de la actividad perjudicial de los genes que contienen repeticiones de nucleótidos extendidas - Google Patents

Agentes nucleósidos para la reducción de la actividad perjudicial de los genes que contienen repeticiones de nucleótidos extendidas

Info

Publication number
CO2017011617A2
CO2017011617A2 CONC2017/0011617A CO2017011617A CO2017011617A2 CO 2017011617 A2 CO2017011617 A2 CO 2017011617A2 CO 2017011617 A CO2017011617 A CO 2017011617A CO 2017011617 A2 CO2017011617 A2 CO 2017011617A2
Authority
CO
Colombia
Prior art keywords
reducing
harmful activity
genes
cell
activity
Prior art date
Application number
CONC2017/0011617A
Other languages
English (en)
Inventor
Stanley N Cohen
Ning Deng
Yanan Feng
Tzu-Hao Cheng
Yun-Yun Wu
Wen-Chieh Hsieh
Original Assignee
Univ Leland Stanford Junior
Univ Nat Yang Ming
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 Univ Leland Stanford Junior, Univ Nat Yang Ming filed Critical Univ Leland Stanford Junior
Publication of CO2017011617A2 publication Critical patent/CO2017011617A2/es

Links

Classifications

    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7042—Compounds having saccharide radicals and heterocyclic rings
    • A61K31/7052—Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides
    • A61K31/706—Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides containing six-membered rings with nitrogen as a ring hetero atom
    • A61K31/7064—Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides containing six-membered rings with nitrogen as a ring hetero atom containing condensed or non-condensed pyrimidines
    • A61K31/7076—Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides containing six-membered rings with nitrogen as a ring hetero atom containing condensed or non-condensed pyrimidines containing purines, e.g. adenosine, adenylic acid
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7042—Compounds having saccharide radicals and heterocyclic rings
    • A61K31/7052—Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides
    • A61K31/706—Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides containing six-membered rings with nitrogen as a ring hetero atom
    • A61K31/7064—Compounds having saccharide radicals and heterocyclic rings having nitrogen as a ring hetero atom, e.g. nucleosides, nucleotides containing six-membered rings with nitrogen as a ring hetero atom containing condensed or non-condensed pyrimidines
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • 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
    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N33/00—Investigating or analysing materials by specific methods not covered by groups G01N1/00 - G01N31/00
    • G01N33/48—Biological material, e.g. blood, urine; Haemocytometers
    • G01N33/50—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
    • G—PHYSICS
    • G01—MEASURING; TESTING
    • G01N—INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIR CHEMICAL OR PHYSICAL PROPERTIES
    • G01N33/00—Investigating or analysing materials by specific methods not covered by groups G01N1/00 - G01N31/00
    • G01N33/48—Biological material, e.g. blood, urine; Haemocytometers
    • G01N33/50—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing
    • G01N33/5005—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving human or animal cells
    • G01N33/5008—Chemical analysis of biological material, e.g. blood, urine; Testing involving biospecific ligand binding methods; Immunological testing involving human or animal cells for testing or evaluating the effect of chemical or biological compounds, e.g. drugs, cosmetics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Molecular Biology (AREA)
  • Biomedical Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Genetics & Genomics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • Epidemiology (AREA)
  • Immunology (AREA)
  • Hematology (AREA)
  • Physics & Mathematics (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Microbiology (AREA)
  • General Engineering & Computer Science (AREA)
  • Urology & Nephrology (AREA)
  • Biochemistry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Cell Biology (AREA)
  • Food Science & Technology (AREA)
  • General Physics & Mathematics (AREA)
  • Pathology (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Analytical Chemistry (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Toxicology (AREA)
  • Tropical Medicine & Parasitology (AREA)

Abstract

Los aspectos de la invención incluyen métodos para la reducción de la actividad perjudicial de un gen objetivo que contiene una repetición de nucleótidos (NR, por sus siglas en inglés) extendida mutante en una célula mediante el contacto de la célula con una cantidad eficaz de un agente nucleósido, así como también composiciones utilizadas en dichos métodos. La actividad perjudicial (por ejemplo, toxicidad y/o disfuncionalidad de los productos codificados por este) de un gen objetivo que contiene NR extendida mutante puede verse reducida en una variedad de diferentes formas, por ejemplo, mediante la reducción (y en algunos casos reducción diferencial, que incluye selectiva) la producción o actividad de los productos de expresión tóxica (por ejemplo, ARN o proteína) codificados por el gen objetivo. Se proporcionan también kits y composiciones para poner en práctica los métodos objeto.
CONC2017/0011617A 2015-05-29 2017-11-15 Agentes nucleósidos para la reducción de la actividad perjudicial de los genes que contienen repeticiones de nucleótidos extendidas CO2017011617A2 (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201562168558P 2015-05-29 2015-05-29
PCT/US2016/033116 WO2016196012A1 (en) 2015-05-29 2016-05-18 Nucleoside agents for the reduction of the deleterious activity of extended nucleotide repeat containing genes

Publications (1)

Publication Number Publication Date
CO2017011617A2 true CO2017011617A2 (es) 2018-02-09

Family

ID=57442140

Family Applications (1)

Application Number Title Priority Date Filing Date
CONC2017/0011617A CO2017011617A2 (es) 2015-05-29 2017-11-15 Agentes nucleósidos para la reducción de la actividad perjudicial de los genes que contienen repeticiones de nucleótidos extendidas

Country Status (16)

Country Link
US (1) US10675293B2 (es)
EP (1) EP3303588A4 (es)
JP (1) JP2018520111A (es)
KR (1) KR20180030461A (es)
CN (1) CN107614686A (es)
AU (1) AU2016270297B2 (es)
BR (1) BR112017022578A2 (es)
CA (1) CA2978521A1 (es)
CL (1) CL2017002608A1 (es)
CO (1) CO2017011617A2 (es)
CR (1) CR20170544A (es)
EA (1) EA201792261A1 (es)
IL (1) IL254304A0 (es)
MX (1) MX2017015302A (es)
SG (1) SG11201707513QA (es)
WO (1) WO2016196012A1 (es)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP6990369B2 (ja) * 2017-05-19 2022-02-03 国立大学法人 熊本大学 遺伝性腎疾患アルポート症候群治療薬に係る評価系
AU2018288771B2 (en) * 2017-06-19 2022-04-14 Thomas W. Sun Compounds for the reduction of the deleterious activity of extended nucleotide repeat containing genes
US10882821B1 (en) 2017-09-26 2021-01-05 The Board Of Trustees Of The Leland Stanford Junior University Enantiomeric compound for the reduction of the deleterious activity of extended nucleotide repeat containing genes
CN112272567A (zh) * 2018-01-22 2021-01-26 战国策智权股份有限公司 提升supt4h在细胞中核苷酸重复序列扩张基因的转录调控能力的方法

Family Cites Families (30)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5968914A (en) * 1987-10-28 1999-10-19 Pro-Neuron, Inc. Treatment of chemotherapeutic agent and antiviral agent toxicity with acylated pyrimidine nucleosides
US5470838A (en) 1987-10-28 1995-11-28 Pro-Neuron, Inc. Method of delivering exogenous uridine or cytidine using acylated uridine or cytidine
JPH09510695A (ja) 1994-01-03 1997-10-28 エイシア・ファーマシュウティカルズ，インコーポレイテッド ８−アザ、６−アザ、および６，８−ジアザ−１，４−ジヒドロキノキサリン−２，３−ジオン類およびそのグリシン／ｎｍｄａリセプターに対するアンタゴニストとしての使用
US6472378B2 (en) 1998-08-31 2002-10-29 Pro-Neuron, Inc. Compositions and methods for treatment of mitochondrial diseases
US7807654B2 (en) 1998-08-31 2010-10-05 Wellstat Therapeutics Corporation Compositions and methods for treatment of mitochondrial diseases
US7915233B1 (en) 1998-08-31 2011-03-29 Wellstat Therapeutics Corporation Compositions and methods for treatment of mitochondrial diseases
NZ513926A (en) 1999-02-23 2001-09-28 Univ California Methods of treatment of mitochondrial disorders
AU1343501A (en) 1999-10-26 2001-05-08 Deltagen, Inc. Transgenic mice containing trp gene disruptions
US20050277133A1 (en) 2001-05-18 2005-12-15 Sirna Therapeutics, Inc. RNA interference mediated treatment of polyglutamine (polyQ) repeat expansion diseases using short interfering nucleic acid (siNA)
US20050096284A1 (en) 2002-02-20 2005-05-05 Sirna Therapeutics, Inc. RNA interference mediated treatment of polyglutamine (polyQ) repeat expansion diseases using short interfering nucleic acid (siNA)
US20080176812A1 (en) 2002-08-05 2008-07-24 Davidson Beverly L Allele-specific silencing of disease genes
WO2004029212A2 (en) 2002-09-25 2004-04-08 University Of Massachusetts In vivo gene silencing by chemically modified and stable sirna
AU2003261405A1 (en) 2002-11-01 2004-06-07 University Of Massachusetts Regulation of transcription elongation factors
EP1599196A4 (en) * 2003-01-17 2006-05-31 Threshold Pharmaceuticals Inc ANTICANCER THERAPY
US20050037992A1 (en) * 2003-07-22 2005-02-17 John Lyons Composition and method for treating neurological disorders
US8680063B2 (en) 2003-09-12 2014-03-25 University Of Massachusetts RNA interference for the treatment of gain-of-function disorders
ES2808561T3 (es) 2003-09-12 2021-03-01 Univ Massachusetts Interferencia por ARN para el tratamiento de trastornos de ganancia de función
WO2006105038A2 (en) 2005-03-25 2006-10-05 The Uab Research Foundation Methods of altering gene expression and methods of treatment utilizing same
EP2526942B1 (en) * 2005-06-08 2017-08-09 The University of North Carolina At Chapel Hill Methods of facilitating neural cell survival using non-peptide and peptide BDNF neurotrophin mimetics
EP1937066A4 (en) 2005-08-18 2008-12-24 Alnylam Pharmaceuticals Inc METHOD AND COMPOSITIONS FOR THE TREATMENT OF NERVOUS DISEASES
US8129357B2 (en) 2006-04-07 2012-03-06 Academia Sinica Method of making and using an adenosine analogue
US7375190B2 (en) 2006-09-19 2008-05-20 National Yang-Ming University Recombinant protein and method of screening for agents that modulate polypeptide aggregation
EP2461819A4 (en) 2009-07-28 2013-07-31 Sangamo Biosciences Inc METHODS AND COMPOSITIONS FOR TREATING TRI-NUCLEOTIDE REPEAT DISORDERS
CN102625809B (zh) 2009-09-11 2015-06-24 Isis制药公司 亨廷顿表达的调节
HUE035188T2 (en) 2009-11-13 2018-05-02 Academia Sinica Adenosine A2A receptor and adenzoin transporter double acting compounds for the prevention and treatment of neurodegenerative diseases
WO2011097641A1 (en) 2010-02-08 2011-08-11 Isis Pharmaceuticals, Inc. Methods and compositions useful in treatment of diseases or conditions related to repeat expansion
US8569254B2 (en) 2010-12-10 2013-10-29 National Yang Ming University Methods for modulating the expression and aggregation of CAG-expanded gene product in cells and methods for identifying agents useful for doing the same
US8618077B2 (en) 2011-04-28 2013-12-31 Emory University Compositions and methods for treating fragile X premutation RVGG repeats-mediated toxicity
IN2014CN03043A (es) 2011-09-30 2015-07-03 Univ Tufts
SG11201603063WA (en) 2013-10-23 2016-05-30 Academia Sinica Compounds for use in prevention and treatment of neurodegenerative diseases and pain

Also Published As

Publication number Publication date
BR112017022578A2 (pt) 2018-07-17
US20180064744A1 (en) 2018-03-08
KR20180030461A (ko) 2018-03-23
US10675293B2 (en) 2020-06-09
IL254304A0 (en) 2017-11-30
JP2018520111A (ja) 2018-07-26
CA2978521A1 (en) 2016-12-08
MX2017015302A (es) 2018-03-28
EP3303588A4 (en) 2019-01-23
AU2016270297A1 (en) 2017-09-21
CN107614686A (zh) 2018-01-19
CL2017002608A1 (es) 2018-06-29
CR20170544A (es) 2018-03-20
EP3303588A1 (en) 2018-04-11
EA201792261A1 (ru) 2018-04-30
SG11201707513QA (en) 2017-10-30
WO2016196012A1 (en) 2016-12-08
AU2016270297B2 (en) 2019-12-12

Similar Documents

Publication Publication Date Title
CO2017011617A2 (es) Agentes nucleósidos para la reducción de la actividad perjudicial de los genes que contienen repeticiones de nucleótidos extendidas
CY1125364T1 (el) Συνθεσεις και μεθοδοι για την αναστολη της εκφρασης του γονιδιου ηαο1 (οξειδαση υδροξυοξεος 1 (γλυκολικη οξειδαση)
MX2020013357A (es) Polimerasas, composiciones y metodos de uso.
MX2020013347A (es) Polimerasas, composiciones y metodos de uso.
BR112016029041A2 (pt) terapia de combinação com inibidores de glutaminase
BR112018010681A8 (pt) método para modificar um sítio alvejado, complexo de enzima de modificação de ácido nucleico, e, ácido nucleico.
EA201691581A1 (ru) Способы и композиции для повышения эффективности направленной модификации генов с применением опосредованной олигонуклеотидами репарации генов
CY1123119T1 (el) Αντινοηματικο νουκλεϊκο οξυ
DOP2019000132A (es) COMPOSICIONES DE ARNi DE SERPINA1 Y SUS MÉTODOS DE USO DE LAS MISMAS.
PE20191351A1 (es) Tratamiento aav de la enfermedad de huntington
MX2020003841A (es) Métodos y composiciones para la producción de nucleósido trifosfatos y ácidos ribonucleicos.
MX2021010668A (es) Proteinas de fusion de citoquinas.
CY1122074T1 (el) Νεα παραγωγα 3'-δεοξυαδενοσινης του φωσφοραμαδικου εστερα toy 2' και/η 5' aminoξεος ως αντικαρκινικες ενωσεις
AR102777A1 (es) COMPOSICIONES DE ARNi CONTRA TMPRSS6 Y MÉTODOS PARA SU USO
CY1122975T1 (el) Συνθεσεις και μεθοδοι για την αναστολη της εκφρασης του γονιδιου alas1
AR088079A1 (es) Metodos para la produccion en sitio de un desinfectante estable para su uso como composicion biocida
MX2015012063A (es) Oligonucleotidos antisentido para el tratamiento de celulas madre del cancer.
BR112017016350A2 (pt) molécula de substrato
MX2019005149A (es) Arn bicatenario con modificaciones quimicas postranscripcionales.
MX2022006748A (es) Composiciones de acidos nucleicos.
PH12018500744A1 (en) Methods of preserving the biological activity of ribonucleic acids
AR105066A1 (es) Enzimas y sistemas crispr
CL2019000920A1 (es) Métodos de preservar la actividad biológica de los ácidos ribonucleicos.
AR110675A1 (es) Oligonucleótidos modificados y métodos de uso
AR125447A2 (es) COMPOSICIONES DE ARNi DE SERPINA1 Y SUS MÉTODOS DE USO