CO2017009083A2 - Arni dirigido a la proteína hungtingtina, partículas víricas que lo contienen y métodos para reducir la toxicicidad del arni - Google Patents

Arni dirigido a la proteína hungtingtina, partículas víricas que lo contienen y métodos para reducir la toxicicidad del arni

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Publication number
CO2017009083A2
CO2017009083A2 CONC2017/0009083A CO2017009083A CO2017009083A2 CO 2017009083 A2 CO2017009083 A2 CO 2017009083A2 CO 2017009083 A CO2017009083 A CO 2017009083A CO 2017009083 A2 CO2017009083 A2 CO 2017009083A2
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guide region
chain
bases
arni
base
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CONC2017/0009083A
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English (en)
Inventor
Lisa M Stanek
Adam Palermo
Brenda Richards
Sergio Pablo Sardi
Catherine O'riordan
Antonius Song
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Genzyme Corp
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Publication of CO2017009083A2 publication Critical patent/CO2017009083A2/es

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Abstract

Se proporcionan en la presente memoria moléculas de ARNi dirigidas a proteínas, ejemplificadas en la proteína hungtingtina (Htt), que comprenden una primera cadena y una segunda cadena, en donde: a) la primera cadena y la segunda cadena forman un dúplex; b) la primera cadena comprende una región guía de al menos 19 bases, en donde la región guía comprende una región semilla que comprende las bases 1-N de la cadena guía, en donde N=7 o N=8; y c) la segunda cadena comprende una región no guía de al menos 19 bases, en donde: (i) la región no guía comprende una secuencia de la protuberancia opuesta a una cualquiera o más de las bases 1-(N+2) de la región guía en el dúplex, en donde opcionalmente la protuberancia es la base 1 o la base N+2 opuesta a la región guía; (ii) la región no guía comprende una secuencia de la protuberancia opuesta a una cualquiera o más de las bases 1-(N+1) de la región guía en el dúplex, en donde opcionalmente la protuberancia es la base 1 o la base N+1 opuesta a la región guía; (iii) la región no guía comprende una secuencia de la protuberancia opuesta a una cualquiera o más de las bases 1-N de la región guía en el dúplex, en donde opcionalmente la protuberancia es la base 1 o la base N opuesta a la región guía. Adicionalmente se proporcionan en la presente memoria casetes de expresión, vectores (p. ej., vectores de rAAV, adenovirales recombinantes, lentivirales recombinantes, y de HSV recombinantes), células, partículas virales, y composiciones farmacéuticas que contienen el ARNi. También se proporcionan un metodo para reducir la toxicidad de un ARNi que comprende introducir una protuberancia en la región no guía del ARNi.
CONC2017/0009083A 2015-02-10 2017-09-05 Arni dirigido a la proteína hungtingtina, partículas víricas que lo contienen y métodos para reducir la toxicicidad del arni CO2017009083A2 (es)

Applications Claiming Priority (2)

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US201562114578P 2015-02-10 2015-02-10
PCT/US2016/017207 WO2016130589A2 (en) 2015-02-10 2016-02-09 VARIANT RNAi

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CO2017009083A2 true CO2017009083A2 (es) 2018-01-31

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US (3) US10450563B2 (es)
EP (1) EP3256588A2 (es)
JP (2) JP2018506304A (es)
KR (1) KR20170110149A (es)
CN (1) CN107438671B (es)
AR (1) AR103646A1 (es)
AU (2) AU2016219396B2 (es)
BR (1) BR112017017028A2 (es)
CA (1) CA2976075A1 (es)
CL (1) CL2017002027A1 (es)
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CR (1) CR20170406A (es)
EA (1) EA201791805A1 (es)
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CN107438671A (zh) 2017-12-05
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WO2016130589A2 (en) 2016-08-18
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CR20170406A (es) 2017-11-14
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AU2016219396B2 (en) 2022-03-17
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EA201791805A1 (ru) 2018-05-31
MX2017010369A (es) 2017-12-14
TN2017000354A1 (en) 2019-01-16
PE20171382A1 (es) 2017-09-15
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MA40819A1 (fr) 2018-07-31
CL2017002027A1 (es) 2018-04-20
US20210047641A1 (en) 2021-02-18
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JP2018506304A (ja) 2018-03-08
AU2016219396A1 (en) 2017-09-28
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US10760079B2 (en) 2020-09-01
MY181458A (en) 2020-12-22
TW201704470A (zh) 2017-02-01
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WO2016130589A3 (en) 2016-09-15
UY36554A (es) 2016-09-30
AR103646A1 (es) 2017-05-24
CA2976075A1 (en) 2016-08-18
PH12017501432A1 (en) 2018-01-15
KR20170110149A (ko) 2017-10-10
TWI781079B (zh) 2022-10-21
US20180023082A1 (en) 2018-01-25
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US20200109401A1 (en) 2020-04-09

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