ES2676600T3 - Fabricación y uso de ARN monocatenario sintetizado in vitro para la introducción en células de mamífero para inducir un efecto biológico o bioquímico - Google Patents

Fabricación y uso de ARN monocatenario sintetizado in vitro para la introducción en células de mamífero para inducir un efecto biológico o bioquímico Download PDF

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ES2676600T3
ES2676600T3 ES16186283T ES16186283T ES2676600T3 ES 2676600 T3 ES2676600 T3 ES 2676600T3 ES 16186283 T ES16186283 T ES 16186283T ES 16186283 T ES16186283 T ES 16186283T ES 2676600 T3 ES2676600 T3 ES 2676600T3
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rna
vitro
stranded rna
induce
biological
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Judith Meis
Anthony D. PERSON
Cynthia S. Chin
Jerome Jendrisak
Gary A. Dahl
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Cellscript Inc
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Cellscript Inc
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    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • A61K48/0066—Manipulation of the nucleic acid to modify its expression pattern, e.g. enhance its duration of expression, achieved by the presence of particular introns in the delivered nucleic acid
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    • A61K48/0008—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
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Abstract

Un procedimiento ex vivo o in vitro para obtener la expresión de al menos una proteína de interés en una célula que comprende: poner en contacto una célula con una composición de ARN que comprende ARN sintetizado in vitro que codifica al menos una proteína de interés, en el que dicha composición de ARN se ha tratado con RNasa III en una solución acuosa tamponada que comprende cationes de magnesio a una concentración de aproximadamente 1-4 mM y una sal que proporciona una fuerza iónica equivalente a al menos aproximadamente 50-300 mM de acetato de potasio o glutamato de potasio, en el que menos del 0,01 % de la masa del ARN de dicha composición de ARN tratado es ARN bicatenario de un tamaño superior a aproximadamente 40 pares de bases de longitud, de modo que dicha al menos una proteína de interés se expresa en dicha célula.
ES16186283T 2011-12-30 2012-12-31 Fabricación y uso de ARN monocatenario sintetizado in vitro para la introducción en células de mamífero para inducir un efecto biológico o bioquímico Active ES2676600T3 (es)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201161582050P 2011-12-30 2011-12-30
US201161582080P 2011-12-30 2011-12-30
US201261651738P 2012-05-25 2012-05-25

Publications (1)

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ES2676600T3 true ES2676600T3 (es) 2018-07-23

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ES16186283T Active ES2676600T3 (es) 2011-12-30 2012-12-31 Fabricación y uso de ARN monocatenario sintetizado in vitro para la introducción en células de mamífero para inducir un efecto biológico o bioquímico
ES18170777T Active ES2770314T3 (es) 2011-12-30 2012-12-31 Fabricación y uso de ARN monocatenario sintetizado in vitro para introducción en células de mamífero para inducir un efecto biológico o bioquímico

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US (4) US10201620B2 (es)
EP (6) EP2798064B1 (es)
AU (5) AU2012362113B2 (es)
DK (3) DK3421601T3 (es)
ES (2) ES2676600T3 (es)
HR (1) HRP20200251T1 (es)
HU (1) HUE047947T2 (es)
LT (2) LT3144389T (es)
PL (2) PL3421601T3 (es)
SI (1) SI3421601T1 (es)
WO (1) WO2013102203A1 (es)

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