Drug repositioning as a therapeutic strategy for neurodegenerations associated with OPA1 mutations

OPA1 mutations are the major cause of dominant optic atrophy (DOA) and the syndromic form DOA plus, pathologies for which there is no established cure. We used a ‘drug repurposing’ approach to identify FDA-approved molecules able to rescue the mitochondrial dysfunctions induced by OPA1 mutations. We...

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Bibliografiske detaljer
Udgivet i:Hum Mol Genet
Main Authors: Aleo, Serena J, Del Dotto, Valentina, Fogazza, Mario, Maresca, Alessandra, Lodi, Tiziana, Goffrini, Paola, Ghelli, Anna, Rugolo, Michela, Carelli, Valerio, Baruffini, Enrico, Zanna, Claudia
Format: Artigo
Sprog:Inglês
Udgivet: Oxford University Press 2020
Fag:
General Article
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC7823107/
https://ncbi.nlm.nih.gov/pubmed/33231680
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1093/hmg/ddaa244
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