CO2018004550A2 - Oligonucleótidos para inducir la expresión paterna de ube3a - Google Patents

Oligonucleótidos para inducir la expresión paterna de ube3a

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Publication number
CO2018004550A2
CO2018004550A2 CONC2018/0004550A CO2018004550A CO2018004550A2 CO 2018004550 A2 CO2018004550 A2 CO 2018004550A2 CO 2018004550 A CO2018004550 A CO 2018004550A CO 2018004550 A2 CO2018004550 A2 CO 2018004550A2
Authority
CO
Colombia
Prior art keywords
ube3a
oligonucleotides
induce
expression
relates
Prior art date
Application number
CONC2018/0004550A
Other languages
English (en)
Inventor
Maj Hedtjärn
Veronica Costa
Marius Hoener
Ravi Jagasia
Mads Aaboe Jensen
Christoph Patsch
Lykke Pedersen
Søren Vestergaard Rasmussen
Original Assignee
Hoffmann La Roche
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 Hoffmann La Roche filed Critical Hoffmann La Roche
Publication of CO2018004550A2 publication Critical patent/CO2018004550A2/es

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Abstract

La presente invención se refiere a oligonucleótidos que son capaces de inducir la expresión de la ubiquitín-proteína ligasa E3A (UBE3A) a partir del alelo parterno en neuronas animales o humanas. Los oligonucleótidos con diana en el supresor del alelo paterno de UBE3A mediante hibridación con el ARN no codificante largo de SNHG14 cadena abajo de SNORD109B. Asimismo, la presente invención se refiere a composiciones y métodos farmacéuticos para el tratamiento del síndrome de Angelman.
CONC2018/0004550A 2015-11-12 2018-04-26 Oligonucleótidos para inducir la expresión paterna de ube3a CO2018004550A2 (es)

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PCT/EP2016/077383 WO2017081223A1 (en) 2015-11-12 2016-11-11 Oligonucleotides for inducing paternal ube3a expression

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