CL2021002120A1 - Métodos y composiciones para inhibir la expresión de cyp27a1. - Google Patents

Métodos y composiciones para inhibir la expresión de cyp27a1.

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Publication number
CL2021002120A1
CL2021002120A1 CL2021002120A CL2021002120A CL2021002120A1 CL 2021002120 A1 CL2021002120 A1 CL 2021002120A1 CL 2021002120 A CL2021002120 A CL 2021002120A CL 2021002120 A CL2021002120 A CL 2021002120A CL 2021002120 A1 CL2021002120 A1 CL 2021002120A1
Authority
CL
Chile
Prior art keywords
cyp27a1
expression
compositions
hepatocytes
methods
Prior art date
Application number
CL2021002120A
Other languages
English (en)
Inventor
Utsav Saxena
Original Assignee
Dicerna Pharmaceuticals Inc
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 Dicerna Pharmaceuticals Inc filed Critical Dicerna Pharmaceuticals Inc
Publication of CL2021002120A1 publication Critical patent/CL2021002120A1/es

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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
    • C12N15/1137—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 enzymes
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    • 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
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • C12N9/0004—Oxidoreductases (1.)
    • C12N9/0071—Oxidoreductases (1.) acting on paired donors with incorporation of molecular oxygen (1.14)
    • C12N9/0077—Oxidoreductases (1.) acting on paired donors with incorporation of molecular oxygen (1.14) with a reduced iron-sulfur protein as one donor (1.14.15)
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12Y114/00—Oxidoreductases acting on paired donors, with incorporation or reduction of molecular oxygen (1.14)
    • C12Y114/15—Oxidoreductases acting on paired donors, with incorporation or reduction of molecular oxygen (1.14) with reduced iron-sulfur protein as one donor, and incorporation of one atom of oxygen (1.14.15)

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  • Virology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
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  • Chemical Kinetics & Catalysis (AREA)
  • Epidemiology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)

Abstract

Esta divulgación se refiere a oligonucleótidos, composiciones y métodos útiles para reducir la expresión de CYP27A1, particularmente en hepatocitos. Los oligonucleótidos divulgados para la reducción de la expresión de CYP27A1 pueden ser bicatenarios o monocatenarios y pueden modificarse para lograr características mejoradas tales como una mayor resistencia a las nucleasas y menor inmunogenicidad. Los oligonucleótidos divulgados para la reducción de la expresión de CYP27A1 también pueden incluir ligandos de direccionamiento para dirigirse a una célula u órgano particular, como los hepatocitos del hígado y pueden usarse para tratar enfermedades hepatobiliares y afecciones relacionadas (por ejemplo, fibrosis hepática).
CL2021002120A 2019-02-12 2021-08-11 Métodos y composiciones para inhibir la expresión de cyp27a1. CL2021002120A1 (es)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US201962804410P 2019-02-12 2019-02-12

Publications (1)

Publication Number Publication Date
CL2021002120A1 true CL2021002120A1 (es) 2022-04-01

Family

ID=69941447

Family Applications (1)

Application Number Title Priority Date Filing Date
CL2021002120A CL2021002120A1 (es) 2019-02-12 2021-08-11 Métodos y composiciones para inhibir la expresión de cyp27a1.

Country Status (13)

Country Link
US (1) US20220186229A1 (es)
EP (1) EP3908661A1 (es)
JP (1) JP2022520653A (es)
KR (1) KR20210132661A (es)
CN (1) CN113692444A (es)
AU (1) AU2020221892A1 (es)
BR (1) BR112021015651A2 (es)
CA (1) CA3128059A1 (es)
CL (1) CL2021002120A1 (es)
IL (1) IL285367A (es)
MX (1) MX2021009754A (es)
SG (1) SG11202108532RA (es)
WO (1) WO2020167593A1 (es)

Family Cites Families (16)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR101215789B1 (ko) 2000-03-30 2012-12-26 화이트헤드 인스티튜트 포 바이오메디칼 리서치 Ｒｎａ 간섭의 ｒｎａ 서열 특이적인 매개체
CZ302719B6 (cs) 2000-12-01 2011-09-21 MAX-PLANCK-Gesellschaft zur Förderung der Wissenschaften e.V. Izolovaná molekula dvouretezcové RNA, zpusob její výroby a její použití
US20050159378A1 (en) 2001-05-18 2005-07-21 Sirna Therapeutics, Inc. RNA interference mediated inhibition of Myc and/or Myb gene expression using short interfering nucleic acid (siNA)
EP1442137A4 (en) 2001-11-07 2005-08-31 Applera Corp UNIVERSAL NUCLEOTIDES FOR NUCLEIC ACID ANALYSIS
US20070265220A1 (en) 2004-03-15 2007-11-15 City Of Hope Methods and compositions for the specific inhibition of gene expression by double-stranded RNA
US20090018097A1 (en) 2005-09-02 2009-01-15 Mdrna, Inc Modification of double-stranded ribonucleic acid molecules
PT2341943T (pt) 2008-09-22 2019-02-06 Dicerna Pharmaceuticals Inc Composições e métodos para a inibição específica de expressão génica por dsrna que possui modificações
EP3067359A1 (en) 2008-09-23 2016-09-14 Scott G. Petersen Self delivering bio-labile phosphate protected pro-oligos for oligonucleotide based therapeutics and mediating rna interference
CN102325534B (zh) 2008-12-18 2016-02-17 戴瑟纳制药公司 延长的dicer酶底物和特异性抑制基因表达的方法
WO2010093788A2 (en) 2009-02-11 2010-08-19 Dicerna Pharmaceuticals, Inc. Multiplex dicer substrate rna interference molecules having joining sequences
WO2011005860A2 (en) 2009-07-07 2011-01-13 Alnylam Pharmaceuticals, Inc. 5' phosphate mimics
WO2011133871A2 (en) 2010-04-22 2011-10-27 Alnylam Pharmaceuticals, Inc. 5'-end derivatives
IN2014CN03749A (es) 2011-10-25 2015-09-25 Isis Pharmaceuticals Inc
KR20150090917A (ko) 2012-12-06 2015-08-06 머크 샤프 앤드 돔 코포레이션 디술피드-차폐 전구약물 조성물 및 방법
AU2015269053A1 (en) 2014-06-06 2016-12-22 Solstice Biologics, Ltd. Polynucleotide constructs having bioreversible and non-bioreversible groups
EP3569711B1 (en) * 2014-12-15 2021-02-03 Dicerna Pharmaceuticals, Inc. Ligand-modified double-stranded nucleic acids

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Publication number Publication date
KR20210132661A (ko) 2021-11-04
IL285367A (en) 2021-09-30
SG11202108532RA (en) 2021-09-29
BR112021015651A2 (pt) 2021-10-05
AU2020221892A1 (en) 2021-08-19
US20220186229A1 (en) 2022-06-16
WO2020167593A1 (en) 2020-08-20
MX2021009754A (es) 2021-09-08
CA3128059A1 (en) 2020-08-20
JP2022520653A (ja) 2022-03-31
CN113692444A (zh) 2021-11-23
EP3908661A1 (en) 2021-11-17

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