CR20180264A - Oligonucléotidos para indicar la expresión paterna de ube3a - Google Patents

Oligonucléotidos para indicar la expresión paterna de ube3a

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Publication number
CR20180264A
CR20180264A CR20180264A CR20180264A CR20180264A CR 20180264 A CR20180264 A CR 20180264A CR 20180264 A CR20180264 A CR 20180264A CR 20180264 A CR20180264 A CR 20180264A CR 20180264 A CR20180264 A CR 20180264A
Authority
CR
Costa Rica
Prior art keywords
ube3a
oligonucléotidos
indicate
expression
oligonucleotides
Prior art date
Application number
CR20180264A
Other languages
English (en)
Inventor
Ravi; Jagasia
Christoph Patsch
Veronica Costa
Søren Vestergaard; Rasmussen
Mads Aaboe; Jensen
Marius; Hoener
Lykke; Pedersen
Maj; Hedtjärn
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 CR20180264A publication Critical patent/CR20180264A/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.
CR20180264A 2015-11-12 2016-11-11 Oligonucléotidos para indicar la expresión paterna de ube3a CR20180264A (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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