AR081295A1 - Tratamiento de enfermedades relacionadas con lim homeobox 2(lhx2) mediante la inhibicion del transcripto antisentido natural a polinucleotidos lhx2 - Google Patents

Tratamiento de enfermedades relacionadas con lim homeobox 2(lhx2) mediante la inhibicion del transcripto antisentido natural a polinucleotidos lhx2

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Publication number
AR081295A1
AR081295A1 ARP110101726A ARP110101726A AR081295A1 AR 081295 A1 AR081295 A1 AR 081295A1 AR P110101726 A ARP110101726 A AR P110101726A AR P110101726 A ARP110101726 A AR P110101726A AR 081295 A1 AR081295 A1 AR 081295A1
Authority
AR
Argentina
Prior art keywords
lhx2
polynucleotide
lim homeobox
oligonucleotide
antisense
Prior art date
Application number
ARP110101726A
Other languages
English (en)
Original Assignee
Opko Curna Llc
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 Opko Curna Llc filed Critical Opko Curna Llc
Publication of AR081295A1 publication Critical patent/AR081295A1/es

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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
    • A61P19/00—Drugs for skeletal disorders
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00—Drugs for disorders of the blood or the extracellular fluid
    • 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
    • C12N2310/111—Antisense spanning the whole gene, or a large part of it
    • 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
    • C12N2310/113—Antisense targeting other non-coding nucleic acids, e.g. antagomirs

Landscapes

  • Health & Medical Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • General Chemical & Material Sciences (AREA)
  • Public Health (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Animal Behavior & Ethology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Biomedical Technology (AREA)
  • Veterinary Medicine (AREA)
  • Biotechnology (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Hematology (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Diabetes (AREA)
  • Biochemistry (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)

Abstract

Reivindicacion 1: Un método de modular una funcion de y/o la expresion de un polinucleotido LIM Homeobox 2 (LHX2) en un sistema biologico que comprende: poner en contacto dicho sistema con al menos un oligonucleotido antisentido de 5 a 30 nucleotidos de longitud en donde dicho al menos un oligonucleotido tiene al menos 50% de identidad de secuencia con un complemento inverso de un antisentido natural de un polinucleotido LIM Homeobox 2 (LHX2); de este modo modula una funcion de y/o la expresion del polinucleotido LIM Homeobox 2 (LHX2). Reivindicacion 2: Un método de modular una funcion de y/o la expresion de un polinucleotido LIM Homeobox 2 (LHX2) en un sistema biologico de acuerdo con la reivindicacion 1 que comprende: poner en contacto dicho sistema biologico con al menos un oligonucleotido antisentido de 5 a 30 nucleotidos de longitud donde dicho al menos un oligonucleotido tiene al menos 50% de identidad de secuencia con un complemento inverso de un polinucleotido que comprende 5 a 30 nucleotidos consecutivos dentro de los nucleotidos de transcripto antisentido natural 1 a 2145 de la SEQ ID Ns: 2, 1 a 606 de la SEQ ID Ns: 3 y 1 a 480 de la SEQ ID Ns: 4; de este modo modula una funcion de y/o la expresion del polinucleotido LIM Homeobox 2 (LHX2). Reivindicacion 18: Un oligonucleotido modificado, sintético que comprende al menos una modificacion donde la al menos una modificacion se selecciona de: al menos un residuo de azucar modificado; al menos una ligadura internucleotídica modificada; al menos un nucleotido modificado, y sus combinaciones; donde dicho oligonucleotido es un compuesto antisentido que se hibrida con y modula la funcion y/o expresion de un gen LIM Homeobox 2 (LHX2)in vivo o in vitro en comparacion con un control normal y donde dicho oligonucleotido tiene al menos 50% de identidad de secuencia con una secuencia de al menos aproximadamente cinco ácidos nucleicos consecutivos que es complementaria con la molécula de ácido nucleico antisentido y/o sentido del polinucleotido LIM Homeobox 2 (LHX2) y alelos, homologos, isoformas, variantes, derivados, mutantes, fragmentos, o sus combinaciones. Reivindicacion 32: Una composicion farmacéutica que comprende uno o más oligonucleotidos específicos para uno o más polinucleotidos LIM Homeobox 2 (LHX2) de acuerdo con la reivindicacion 18 y un excipiente farmacéuticamente aceptable. Reivindicacion 37: Un método de prevenir o tratar una enfermedad asociada con al menos un polinucleotido LIM Homeobox 2 (LHX2) y/o al menos un producto codificado de este, que comprende: administrar a un paciente una dosis terapéuticamente efectiva de al menos un oligonucleotido antisentido que se une a una secuencia antisentido natural de dicho al menos un polinucleotido LIM Homeobox 2 (LHX2) y modula la expresion de dicho al menos un polinucleotido LIM Homeobox 2 (LHX2); y de este modo prevenir o tratar la enfermedad asociada con el al menos un polinucleotido LIM Homeobox 2 (LHX2) y/o al menos un producto codificado de este.
ARP110101726A 2010-05-19 2011-05-19 Tratamiento de enfermedades relacionadas con lim homeobox 2(lhx2) mediante la inhibicion del transcripto antisentido natural a polinucleotidos lhx2 AR081295A1 (es)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US34628410P 2010-05-19 2010-05-19

Publications (1)

Publication Number Publication Date
AR081295A1 true AR081295A1 (es) 2012-08-01

Family

ID=44992329

Family Applications (1)

Application Number Title Priority Date Filing Date
ARP110101726A AR081295A1 (es) 2010-05-19 2011-05-19 Tratamiento de enfermedades relacionadas con lim homeobox 2(lhx2) mediante la inhibicion del transcripto antisentido natural a polinucleotidos lhx2

Country Status (3)

Country Link
AR (1) AR081295A1 (es)
TW (1) TW201143782A (es)
WO (1) WO2011146675A2 (es)

Families Citing this family (13)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3702460A1 (en) 2010-11-12 2020-09-02 The General Hospital Corporation Polycomb-associated non-coding rnas
US9920317B2 (en) 2010-11-12 2018-03-20 The General Hospital Corporation Polycomb-associated non-coding RNAs
US10837014B2 (en) 2012-05-16 2020-11-17 Translate Bio Ma, Inc. Compositions and methods for modulating SMN gene family expression
US10174315B2 (en) 2012-05-16 2019-01-08 The General Hospital Corporation Compositions and methods for modulating hemoglobin gene family expression
WO2013173598A1 (en) 2012-05-16 2013-11-21 Rana Therapeutics, Inc. Compositions and methods for modulating atp2a2 expression
WO2013173652A1 (en) 2012-05-16 2013-11-21 Rana Therapeutics, Inc. Compositions and methods for modulating gene expression
EP2850190B1 (en) 2012-05-16 2020-07-08 Translate Bio MA, Inc. Compositions and methods for modulating mecp2 expression
WO2013173645A1 (en) 2012-05-16 2013-11-21 Rana Therapeutics, Inc. Compositions and methods for modulating utrn expression
DK2850186T3 (en) 2012-05-16 2019-04-08 Translate Bio Ma Inc COMPOSITIONS AND PROCEDURES FOR MODULATING SMN GENFAMILY EXPRESSION
US10174328B2 (en) 2013-10-04 2019-01-08 Translate Bio Ma, Inc. Compositions and methods for treating amyotrophic lateral sclerosis
CA2966044A1 (en) 2014-10-30 2016-05-06 The General Hospital Corporation Methods for modulating atrx-dependent gene repression
WO2016149455A2 (en) 2015-03-17 2016-09-22 The General Hospital Corporation The rna interactome of polycomb repressive complex 1 (prc1)
CN114917367A (zh) * 2021-06-02 2022-08-19 中国科学院动物研究所 Lhx2在促进中枢系统神经元的损伤修复再生中的应用

Also Published As

Publication number Publication date
WO2011146675A2 (en) 2011-11-24
WO2011146675A3 (en) 2012-03-08
TW201143782A (en) 2011-12-16

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