Angelman syndrome patient neuron screen identifies a potent and selective clinical ASO targeting UBE...
Angelman syndrome patient neuron screen identifies a potent and selective clinical ASO targeting UBE3A-ATS with long lasting effect in cynomolgus monkey
About this item
Full title
Author / Creator
Jagasia, Ravi , Bon, Charlotte , Rasmussen, Soren V , Badillo, Solveig , Tehler, Disa , Buchy, Daniele , Berrera, Marco , Prasad, Megana , Terrigno, Marco , Pandya, Nikhil J , Costa, Veronica , Wang, Congwei , Pedersen, Lykke , Miller, Meghan T , Erichsen, Kamille Dumong , Joenson, Lars , Hipp, Joerg , Azad Bonni , Mueller, Lutz , Braendli-Baiocco, Annamaria , Kremer, Thomas , Koller, Erich and Hoener, Marius
Publisher
Cold Spring Harbor: Cold Spring Harbor Laboratory Press
Journal title
Language
English
Formats
Publication information
Publisher
Cold Spring Harbor: Cold Spring Harbor Laboratory Press
Subjects
More information
Scope and Contents
Contents
Angelman syndrome (AS) is a severe neurodevelopmental disorder caused by the loss of neuronal E3 ligase UBE3A with no available treatment. Restoring UBE3A levels via downregulation of the paternally cis-acting long non-coding antisense transcript (UBE3A-ATS) is a potentially disease modifying. Developing molecules targeting human UBE3A-ATS is chall...
Alternative Titles
Full title
Angelman syndrome patient neuron screen identifies a potent and selective clinical ASO targeting UBE3A-ATS with long lasting effect in cynomolgus monkey
Authors, Artists and Contributors
Author / Creator
Bon, Charlotte
Rasmussen, Soren V
Badillo, Solveig
Tehler, Disa
Buchy, Daniele
Berrera, Marco
Prasad, Megana
Terrigno, Marco
Pandya, Nikhil J
Costa, Veronica
Wang, Congwei
Pedersen, Lykke
Miller, Meghan T
Erichsen, Kamille Dumong
Joenson, Lars
Hipp, Joerg
Azad Bonni
Mueller, Lutz
Braendli-Baiocco, Annamaria
Kremer, Thomas
Koller, Erich
Hoener, Marius
Identifiers
Primary Identifiers
Record Identifier
TN_cdi_proquest_journals_2675432834
Permalink
https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_proquest_journals_2675432834
Other Identifiers
E-ISSN
2692-8205
DOI
10.1101/2022.06.09.495066
How to access this item
https://www.proquest.com/docview/2675432834?pq-origsite=primo&accountid=13902
