MX2017015302A - Agentes nucleosidos para la reduccion de la actividad perjudicial de los genes que contienen repeticiones de nucleotidos extendidas. - Google Patents
Agentes nucleosidos para la reduccion de la actividad perjudicial de los genes que contienen repeticiones de nucleotidos extendidas.Info
- Publication number
- MX2017015302A MX2017015302A MX2017015302A MX2017015302A MX2017015302A MX 2017015302 A MX2017015302 A MX 2017015302A MX 2017015302 A MX2017015302 A MX 2017015302A MX 2017015302 A MX2017015302 A MX 2017015302A MX 2017015302 A MX2017015302 A MX 2017015302A
- Authority
- MX
- Mexico
- Prior art keywords
- activity
- nucleotide repeat
- reduction
- reducing
- target gene
- Prior art date
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
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.
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 |
---|---|
MX2017015302A true MX2017015302A (es) | 2018-03-28 |
Family
ID=57442140
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
MX2017015302A MX2017015302A (es) | 2015-05-29 | 2016-05-18 | Agentes nucleosidos para la reduccion de la actividad perjudicial de los genes que contienen repeticiones de nucleotidos 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)
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 |
TWI796420B (zh) * | 2018-01-22 | 2023-03-21 | 國立陽明交通大學 | 提升supt4h在細胞中核苷酸重複序列擴張基因之轉錄調控能力的方法 |
Family Cites Families (30)
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US5470838A (en) | 1987-10-28 | 1995-11-28 | Pro-Neuron, Inc. | Method of delivering exogenous uridine or cytidine using acylated uridine or cytidine |
US5968914A (en) * | 1987-10-28 | 1999-10-19 | Pro-Neuron, Inc. | Treatment of chemotherapeutic agent and antiviral agent toxicity with acylated pyrimidine nucleosides |
US5620978A (en) | 1994-01-03 | 1997-04-15 | State Of Oregon, Acting By And Through The Oregon State Board Of Higher Education, Acting For And On Behalf Of The Oregon Health Sciences University And The University Of Oregon, Eugene Oregon | 8-aza, 6-aza and 6,8-diaza-1,4-dihydroquinoxaline-2,3-diones and the use thereof as antagonists for the glycine/NMDA receptor |
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 |
PT1171137E (pt) | 1999-02-23 | 2008-03-17 | Univ California | Utilização de triacetiluridina para o tratamento de perturbações mitocondriais |
EP1224199A1 (en) | 1999-10-26 | 2002-07-24 | 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 |
CA2500224C (en) | 2002-09-25 | 2015-04-28 | University Of Massachusetts | In vivo gene silencing by chemically modified and stable sirna |
US20040152651A1 (en) | 2002-11-01 | 2004-08-05 | Rana Tariq M. | Regulation of transcription elongation factors |
JP2006515885A (ja) * | 2003-01-17 | 2006-06-08 | スレッシュオールド ファーマシューティカルズ, インコーポレイテッド | 癌の処置のための併用療法 |
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 |
DK2821085T3 (da) | 2003-09-12 | 2020-08-03 | Univ Massachusetts | Rna-interferens til behandling af "gain-of-function"-forstyrrelser |
WO2006105038A2 (en) | 2005-03-25 | 2006-10-05 | The Uab Research Foundation | Methods of altering gene expression and methods of treatment utilizing same |
JP5461009B2 (ja) | 2005-06-08 | 2014-04-02 | ザ ユニバーシティ オブ ノース カロライナ アット チャペル ヒル | 非ペプチド及びペプチドbdnfニューロトロフィン類似体を用いた神経細胞生存促進法 |
WO2007022470A2 (en) | 2005-08-18 | 2007-02-22 | Alnylam Pharmaceuticals, Inc. | Methods and compositions for treating neurological disease |
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 |
US9234016B2 (en) | 2009-07-28 | 2016-01-12 | Sangamo Biosciences, Inc. | Engineered zinc finger proteins for treating trinucleotide repeat disorders |
HUE033113T2 (en) | 2009-09-11 | 2017-11-28 | Ionis Pharmaceuticals Inc | Modifying Huntingtin expression |
US20120295863A1 (en) * | 2009-11-13 | 2012-11-22 | Yun-Lian Lin | Dual-Action Compounds Targeting Adenosine A2A Receptor and Adenosine Transporter for Prevention and Treatment of Neurodegenerative Diseases |
US20130059902A1 (en) | 2010-02-08 | 2013-03-07 | 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 |
CA2850367C (en) | 2011-09-30 | 2021-06-01 | Tufts University | Uridine diphosphate derivatives, compositions and methods for treating neurodegenerative disorders |
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-
2016
- 2016-05-18 BR BR112017022578-6A patent/BR112017022578A2/pt not_active IP Right Cessation
- 2016-05-18 CR CR20170544A patent/CR20170544A/es unknown
- 2016-05-18 MX MX2017015302A patent/MX2017015302A/es unknown
- 2016-05-18 SG SG11201707513QA patent/SG11201707513QA/en unknown
- 2016-05-18 AU AU2016270297A patent/AU2016270297B2/en not_active Ceased
- 2016-05-18 EP EP16803979.0A patent/EP3303588A4/en not_active Withdrawn
- 2016-05-18 CN CN201680025759.2A patent/CN107614686A/zh active Pending
- 2016-05-18 CA CA2978521A patent/CA2978521A1/en not_active Abandoned
- 2016-05-18 US US15/554,679 patent/US10675293B2/en active Active
- 2016-05-18 KR KR1020177030679A patent/KR20180030461A/ko unknown
- 2016-05-18 JP JP2017560913A patent/JP2018520111A/ja active Pending
- 2016-05-18 WO PCT/US2016/033116 patent/WO2016196012A1/en active Application Filing
- 2016-05-18 EA EA201792261A patent/EA201792261A1/ru unknown
-
2017
- 2017-09-04 IL IL254304A patent/IL254304A0/en unknown
- 2017-10-13 CL CL2017002608A patent/CL2017002608A1/es unknown
- 2017-11-15 CO CONC2017/0011617A patent/CO2017011617A2/es unknown
Also Published As
Publication number | Publication date |
---|---|
EP3303588A1 (en) | 2018-04-11 |
EP3303588A4 (en) | 2019-01-23 |
EA201792261A1 (ru) | 2018-04-30 |
AU2016270297B2 (en) | 2019-12-12 |
CR20170544A (es) | 2018-03-20 |
CN107614686A (zh) | 2018-01-19 |
WO2016196012A1 (en) | 2016-12-08 |
JP2018520111A (ja) | 2018-07-26 |
SG11201707513QA (en) | 2017-10-30 |
KR20180030461A (ko) | 2018-03-23 |
IL254304A0 (en) | 2017-11-30 |
CO2017011617A2 (es) | 2018-02-09 |
US20180064744A1 (en) | 2018-03-08 |
CL2017002608A1 (es) | 2018-06-29 |
AU2016270297A1 (en) | 2017-09-21 |
CA2978521A1 (en) | 2016-12-08 |
BR112017022578A2 (pt) | 2018-07-17 |
US10675293B2 (en) | 2020-06-09 |
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