MX2020005680A - Tratamiento antisentido del sindrome de angelman. - Google Patents

Tratamiento antisentido del sindrome de angelman.

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Publication number
MX2020005680A
MX2020005680A MX2020005680A MX2020005680A MX2020005680A MX 2020005680 A MX2020005680 A MX 2020005680A MX 2020005680 A MX2020005680 A MX 2020005680A MX 2020005680 A MX2020005680 A MX 2020005680A MX 2020005680 A MX2020005680 A MX 2020005680A
Authority
MX
Mexico
Prior art keywords
angelman syndrome
ube3a
antisense treatment
disclosed
treatment
Prior art date
Application number
MX2020005680A
Other languages
English (en)
Inventor
Scott Victor Dindot
Original Assignee
Texas A & M Univ Sys
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 Texas A & M Univ Sys filed Critical Texas A & M Univ Sys
Publication of MX2020005680A publication Critical patent/MX2020005680A/es

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    • 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
    • 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
    • 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
    • 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
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • C07H21/02—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with ribosyl as saccharide radical
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • C07H21/04—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with deoxyribosyl as saccharide radical
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications

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

Abstract

En la presente descripción se describen oligonucleótidos antisentido que son capaces de inducir la expresión de la proteína ubiquitina ligasa E3A (UBE3A) a partir del alelo paterno en neuronas animales o humanas; los oligonucleótidos se dirigen al supresor del alelo paterno de UBE3A por hibridación al ARN no codificante largo de SNHG14 en el extremo 5' de UBE3A-AS, que está aguas abajo del ARNpn SNORDII5-45. Además, se describen composiciones farmacéuticas y métodos para el tratamiento del síndrome de Angelman.
MX2020005680A 2017-12-01 2018-11-30 Tratamiento antisentido del sindrome de angelman. MX2020005680A (es)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201762593431P 2017-12-01 2017-12-01
US201862676034P 2018-05-24 2018-05-24
PCT/US2018/063416 WO2019109001A1 (en) 2017-12-01 2018-11-30 Angelman syndrome antisense treatment

Publications (1)

Publication Number Publication Date
MX2020005680A true MX2020005680A (es) 2020-08-20

Family

ID=66664231

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2020005680A MX2020005680A (es) 2017-12-01 2018-11-30 Tratamiento antisentido del sindrome de angelman.

Country Status (16)

Country Link
US (4) US11198869B2 (es)
EP (2) EP3717646A4 (es)
JP (2) JP7297320B2 (es)
KR (1) KR20200094152A (es)
CN (1) CN111433361B (es)
AU (1) AU2018375807A1 (es)
BR (1) BR112020009431A2 (es)
CA (1) CA3079755A1 (es)
CL (1) CL2020001344A1 (es)
CO (1) CO2020006361A2 (es)
IL (1) IL274146A (es)
MX (1) MX2020005680A (es)
PE (1) PE20211238A1 (es)
RU (1) RU2020121752A (es)
SG (1) SG11202003596WA (es)
WO (1) WO2019109001A1 (es)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CR20220077A (es) 2015-11-12 2023-04-11 Hoffmann La Roche Oligonucleótidos para inducir la expresión paterna de ube3a (divisional 2020-118)
CN117431244A (zh) 2019-03-29 2024-01-23 Ionis制药公司 用于调节ube3a-ats的化合物和方法
WO2022076415A1 (en) * 2020-10-06 2022-04-14 University Of Maryland, Baltimore Rapid diagnostic electrochemical biosensing targeted with antisense oligonucleotides
WO2023172535A2 (en) * 2022-03-07 2023-09-14 University Of Connecticut shRNA TARGETING SNORD115 LOCATIONS TO RESTORE PATERNAL UBE3A GENE EXPRESSION IN ANGELMAN SYNDROME

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Also Published As

Publication number Publication date
EP4328306A2 (en) 2024-02-28
CL2020001344A1 (es) 2020-09-21
EP3717646A1 (en) 2020-10-07
KR20200094152A (ko) 2020-08-06
WO2019109001A1 (en) 2019-06-06
EP3717646A4 (en) 2021-12-01
US11685920B2 (en) 2023-06-27
JP2023123517A (ja) 2023-09-05
US20230332152A1 (en) 2023-10-19
SG11202003596WA (en) 2020-05-28
US11198869B2 (en) 2021-12-14
CO2020006361A2 (es) 2020-08-10
JP7297320B2 (ja) 2023-06-26
CA3079755A1 (en) 2019-06-06
BR112020009431A2 (pt) 2020-10-13
US11739328B2 (en) 2023-08-29
US20230112629A1 (en) 2023-04-13
JP2021505129A (ja) 2021-02-18
CN111433361A (zh) 2020-07-17
US20200370046A1 (en) 2020-11-26
CN111433361B (zh) 2024-03-29
AU2018375807A1 (en) 2020-05-07
US20220090075A1 (en) 2022-03-24
PE20211238A1 (es) 2021-07-09
RU2020121752A (ru) 2021-12-30
IL274146A (en) 2020-06-30

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