An orally available, brain penetrant, small molecule lowers huntingtin levels by enhancing pseudoexo...
An orally available, brain penetrant, small molecule lowers huntingtin levels by enhancing pseudoexon inclusion
About this item
Full title
Author / Creator
Keller, Caroline Gubser , Shin, Youngah , Monteys, Alex Mas , Renaud, Nicole , Beibel, Martin , Teider, Natalia , Peters, Thomas , Faller, Thomas , St-Cyr, Sophie , Knehr, Judith , Roma, Guglielmo , Reyes, Alejandro , Hild, Marc , Lukashev, Dmitriy , Theil, Diethilde , Dales, Natalie , Cha, Jang-Ho , Borowsky, Beth , Dolmetsch, Ricardo , Davidson, Beverly L. and Sivasankaran, Rajeev
Publisher
London: Nature Publishing Group UK
Journal title
Language
English
Formats
Publication information
Publisher
London: Nature Publishing Group UK
Subjects
More information
Scope and Contents
Contents
Huntington’s Disease (HD) is a progressive neurodegenerative disorder caused by CAG trinucleotide repeat expansions in exon 1 of the huntingtin (
HTT
) gene. The mutant HTT (mHTT) protein causes neuronal dysfunction, causing progressive motor, cognitive and behavioral abnormalities. Current treatments for HD only alleviate symptoms, but cereb...
Alternative Titles
Full title
An orally available, brain penetrant, small molecule lowers huntingtin levels by enhancing pseudoexon inclusion
Authors, Artists and Contributors
Author / Creator
Shin, Youngah
Monteys, Alex Mas
Renaud, Nicole
Beibel, Martin
Teider, Natalia
Peters, Thomas
Faller, Thomas
St-Cyr, Sophie
Knehr, Judith
Roma, Guglielmo
Reyes, Alejandro
Hild, Marc
Lukashev, Dmitriy
Theil, Diethilde
Dales, Natalie
Cha, Jang-Ho
Borowsky, Beth
Dolmetsch, Ricardo
Davidson, Beverly L.
Sivasankaran, Rajeev
Identifiers
Primary Identifiers
Record Identifier
TN_cdi_doaj_primary_oai_doaj_org_article_917384fbd2e34e4887d645320e87dd19
Permalink
https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_doaj_primary_oai_doaj_org_article_917384fbd2e34e4887d645320e87dd19
Other Identifiers
ISSN
2041-1723
E-ISSN
2041-1723
DOI
10.1038/s41467-022-28653-6
