Article
Efficient Neuroprotective Rescue of Sacsin-Related Disease Phenotypes in Zebrafish.
International journal of molecular sciences - 5 Aug 2021
Naef Valentina, Marchese Maria, Ogi Asahi, Fichi Gianluca, Galatolo Daniele, Licitra Rosario, Doccini Stefano, Verri Tiziano, Argenton Francesco, Morani Federica, Santorelli Filippo M
Abstract excerpt
Autosomal recessive spastic ataxia of Charlevoix-Saguenay (ARSACS) is a multisystem hereditary ataxia associated with mutations in SACS, which encodes sacsin, a protein of still only partially understood function. Although mouse models of ARSACS mimic largely the disease progression seen in humans, their use in the validation of effective therapies has not yet been proposed. Recently, the teleost Danio rerio has...
Read the complete abstract on PubMedTopics
Share this publication in a Topic to start or enrich a Post.
