AR081398A1 - Tratamiento de enfermedades relacionadas con par4 mediante inhibicion de transcripto antisentido natural a par4 - Google Patents

Tratamiento de enfermedades relacionadas con par4 mediante inhibicion de transcripto antisentido natural a par4

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Publication number
AR081398A1
AR081398A1 ARP110101675A ARP110101675A AR081398A1 AR 081398 A1 AR081398 A1 AR 081398A1 AR P110101675 A ARP110101675 A AR P110101675A AR P110101675 A ARP110101675 A AR P110101675A AR 081398 A1 AR081398 A1 AR 081398A1
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AR
Argentina
Prior art keywords
par4
oligonucleotide
polynucleotide
antisense
expression
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ARP110101675A
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English (en)
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Opko Curna Llc
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Publication of AR081398A1 publication Critical patent/AR081398A1/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
    • C12N15/1138—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 receptors or cell surface proteins
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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
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    • Y02—TECHNOLOGIES OR APPLICATIONS FOR MITIGATION OR ADAPTATION AGAINST CLIMATE CHANGE
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    • Y02A50/30—Against vector-borne diseases, e.g. mosquito-borne, fly-borne, tick-borne or waterborne diseases whose impact is exacerbated by climate change

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Abstract

Reivindicacion 1: Uso de por lo menos un oligonucleotido antisentido de 5 a 30 nucleotidos de largo en la manufactura de un medicamento para modular una funcion y/o la expresion de un polinucleotido de PAR4 en un sistema biologico, donde dicho por lo menos un oligonucleotido tiene por lo menos 50% de identidad de secuencia con un complemento inverso de un antisentido natural de un polinucleotido de PAR4. Reivindicacion 2: El uso de acuerdo con la reivindicacion 1, donde dicho por lo menos un oligonucleotido tiene por lo menos 50% de identidad de secuencia con un complemento inverso de un polinucleotido que comprende de 5 a 30 nucleotidos consecutivos dentro de los nucleotidos del transcrito antisentido natural 1 a 354 de SEC ID Ns: 2. Reivindicacion 18: Un oligonucleotido sintético modificado que comprende por lo menos una modificacion donde la por lo menos una modificacion se selecciona entre: por lo menos un resto azucar modificado; por lo menos una ligacion internucleotídica modificada; por lo menos un nucleotido modificado y sus combinaciones; donde dicho oligonucleotido es un compuesto antisentido el cual se hibrida a y modula la funcion y/o expresion de un gen de PAR4 in vivo o in vitro en comparacion con un control normal y donde dicho oligonucleotido tiene por lo menos 50% de identidad de secuencia con una secuencia de por lo menos aproximadamente cinco ácidos nucleicos consecutivos que es complementaria a la molécula de ácido nucleico antisentido y/o sentido del polinucleotido de PAR4 y los alelos, homologos, isoformas, variantes, derivados, mutantes, fragmentos, o combinaciones de los mismos. Reivindicacion 32: Una composicion farmacéutica que comprende uno o más oligonucleotidos específicos para uno o más polinucleotidos de PAR4 de acuerdo con la reivindicacion 18 y un excipiente farmacéuticamente aceptable. Reivindicacion 38: El uso de acuerdo con la reivindicacion 37, donde una enfermedad asociada con el por lo menos un polinucleotido de PAR4 se selecciona entre: una enfermedad o trastorno asociado con la funcion y/o expresion anormal de PAR4, cáncer, apoptosis aberrante, una enfermedad o trastorno proliferativo, una enfermedad o trastorno cardiovascular, una enfermedad o trastorno inflamatorio, una enfermedad o trastorno neurologico y envejecimiento. Reivindicacion 46: Un método in vitro para modular una funcion de y/o la expresion de un polinucleotido de PAR4 en células o tejidos de un paciente que comprende: poner en contacto dichas células o tejidos con por lo menos un oligonucleotido antisentido de 5 a 30 nucleotidos de largo donde dicho oligonucleotido tiene por lo menos 50% de identidad de secuencia con un oligonucleotido antisentido al polinucleotido de PAR4; modulando de ese modo una funcion de y/o la expresion del polinucleotido de PAR4 en células o tejidos del paciente. Reivindicacion 58: Un método in vitro para modular una funcion de y/o la expresion de un gen de PAR4 en células o tejidos de mamífero que comprende: poner en contacto dichas células o tejidos con por lo menos un oligonucleotido de ARN corto de interferencia (siRNA) de 5 a 30 nucleotidos de largo, siendo dicho por lo menos un oligonucleotido de siRNA específico para un polinucleotido antisentido de un polinucleotido de PAR4, donde dicho por lo menos un oligonucleotido de siRNA tiene por lo menos 50% de identidad de secuencia con una secuencia complementaria de por lo menos aproximadamente cinco ácidos nucleicos consecutivos de la molécula de ácido nucleico antisentido y/o sentido del polinucleotido de PAR4; y, modular una funcion de y/o la expresion de PAR4 en células o tejidos de mamíferos.
ARP110101675A 2010-05-14 2011-05-16 Tratamiento de enfermedades relacionadas con par4 mediante inhibicion de transcripto antisentido natural a par4 AR081398A1 (es)

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US (3) US8980857B2 (es)
EP (1) EP2569432B1 (es)
JP (1) JP5982362B2 (es)
KR (1) KR101844091B1 (es)
CN (1) CN102985543B (es)
AR (1) AR081398A1 (es)
CA (1) CA2798401C (es)
DK (1) DK2569432T3 (es)
ES (1) ES2671901T3 (es)
NO (1) NO2569432T3 (es)
SA (1) SA111320451B1 (es)
TW (2) TWI531370B (es)
WO (1) WO2011143640A2 (es)

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KR20150030205A (ko) 2012-05-16 2015-03-19 라나 테라퓨틱스, 인크. Smn 유전자 패밀리 발현을 조절하기 위한 조성물 및 방법
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EP3271460A4 (en) 2015-03-17 2019-03-13 The General Hospital Corporation INTERACTOME RNA OF COMPLEX REPRESSIVE POLYCOMB 1 (PRC1)
CN110290794A (zh) 2016-11-01 2019-09-27 纽约州州立大学研究基金会 5-卤代尿嘧啶修饰的微rna及其在癌症治疗中的用途
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