Dimethyl fumarate dosing in humans increases frataxin expression: A potential therapy for Friedreich's Ataxia.
Friedreich's Ataxia (FA) is an inherited neurodegenerative disorder resulting from decreased expression of the mitochondrial protein frataxin, for which there is no approved therapy. High throughput screening of clinically used drugs identified Dimethyl fumarate (DMF) as protective in FA patien...
Main Authors: | Mittal Jasoliya, Francesco Sacca, Sunil Sahdeo, Frederic Chedin, Chiara Pane, Vincenzo Brescia Morra, Alessandro Filla, Mark Pook, Gino Cortopassi |
---|---|
Format: | Article |
Language: | English |
Published: |
Public Library of Science (PLoS)
2019-01-01
|
Series: | PLoS ONE |
Online Access: | https://doi.org/10.1371/journal.pone.0217776 |
Similar Items
-
Epigenetic modification of the frataxin gene for the treatment of Friedreich’s ataxia
by: Vilema Enriquez, MG
Published: (2018) -
Novel frataxin isoforms may contribute to the pathological mechanism of Friedreich ataxia.
by: Haiyan Xia, et al.
Published: (2012-01-01) -
DNA methylation in Friedreich ataxia silences expression of frataxin isoform E
by: Layne N. Rodden, et al.
Published: (2022-03-01) -
Simultaneous Quantification of Mitochondrial Mature Frataxin and Extra-Mitochondrial Frataxin Isoform E in Friedreich’s Ataxia Blood
by: Qingqing Wang, et al.
Published: (2022-04-01) -
Strategies for increasing frataxin expression in Friedreich's ataxia: modulation of the epigenome and proteome
by: Quinlan, R
Published: (2021)