BR112018007633A2 - oligonucleotídeos para indução de expressão de ube3a paterna - Google Patents

oligonucleotídeos para indução de expressão de ube3a paterna

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Publication number
BR112018007633A2
BR112018007633A2 BR112018007633-3A BR112018007633A BR112018007633A2 BR 112018007633 A2 BR112018007633 A2 BR 112018007633A2 BR 112018007633 A BR112018007633 A BR 112018007633A BR 112018007633 A2 BR112018007633 A2 BR 112018007633A2
Authority
BR
Brazil
Prior art keywords
oligonucleotides
expression induction
paternal ube3a
ube3a
ube3a expression
Prior art date
Application number
BR112018007633-3A
Other languages
English (en)
Inventor
COSTA Veronica
Hedtjärn Maj
Hoener Marius
Jagasia Ravi
Aaboe Jensen Mads
Patsch Christoph
PEDERSEN Lykke
Vestergaard Rasmussen Søren
Original Assignee
F. Hoffmann-La Roche Ag
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 F. Hoffmann-La Roche Ag filed Critical F. Hoffmann-La Roche Ag
Publication of BR112018007633A2 publication Critical patent/BR112018007633A2/pt

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Abstract

a presente invenção refere-se a oligonucleotídeos que são capazes de indução da expressão de proteína ubiquitina ligase e3a (ube3a) a partir do alelo paterno em neurônios animais ou humanos. os oligonucleotídeos se direcionam ao supressor do alelo paterno de ube3a através da hibridização para rna de não codificação longo de snhg14 a jusante de snord109b. a presente invenção refere-se ainda a composições farmacêuticas e métodos para tratamento de síndrome de angelman.
BR112018007633-3A 2015-11-12 2016-11-11 oligonucleotídeos para indução de expressão de ube3a paterna BR112018007633A2 (pt)

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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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