High-content screening identifies a small molecule that restores AP-4-dependent protein trafficking...
High-content screening identifies a small molecule that restores AP-4-dependent protein trafficking in neuronal models of AP-4-associated hereditary spastic paraplegia
About this item
Full title
Author / Creator
Saffari, Afshin , Brechmann, Barbara , Böger, Cedric , Saber, Wardiya Afshar , Jumo, Hellen , Whye, Dosh , Wood, Delaney , Wahlster, Lara , Alecu, Julian E. , Ziegler, Marvin , Scheffold, Marlene , Winden, Kellen , Hubbs, Jed , Buttermore, Elizabeth D. , Barrett, Lee , Borner, Georg H. H. , Davies, Alexandra K. , Ebrahimi-Fakhari, Darius and Sahin, Mustafa
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
Unbiased phenotypic screens in patient-relevant disease models offer the potential to detect therapeutic targets for rare diseases. In this study, we developed a high-throughput screening assay to identify molecules that correct aberrant protein trafficking in adapter protein complex 4 (AP-4) deficiency, a rare but prototypical form of childhood-on...
Alternative Titles
Full title
High-content screening identifies a small molecule that restores AP-4-dependent protein trafficking in neuronal models of AP-4-associated hereditary spastic paraplegia
Authors, Artists and Contributors
Author / Creator
Brechmann, Barbara
Böger, Cedric
Saber, Wardiya Afshar
Jumo, Hellen
Whye, Dosh
Wood, Delaney
Wahlster, Lara
Alecu, Julian E.
Ziegler, Marvin
Scheffold, Marlene
Winden, Kellen
Hubbs, Jed
Buttermore, Elizabeth D.
Barrett, Lee
Borner, Georg H. H.
Davies, Alexandra K.
Ebrahimi-Fakhari, Darius
Sahin, Mustafa
Identifiers
Primary Identifiers
Record Identifier
TN_cdi_doaj_primary_oai_doaj_org_article_b52e18bceed849439f7ec10f0de5714b
Permalink
https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_doaj_primary_oai_doaj_org_article_b52e18bceed849439f7ec10f0de5714b
Other Identifiers
ISSN
2041-1723
E-ISSN
2041-1723
DOI
10.1038/s41467-023-44264-1
