Communicated by the Degenerative Processes, Stress and Aging Team
The expression of a mutated toxic protein (Ataxin3-78Q – SCA3) in fly photoreceptors leads to a progressive degeneration of the retina (SCA3 + control early stage vs. SCA3 + control late stage). This phenotype is rescued when the transport protein KPNA3 is down regulated (KPNA3 KD).
“Targeting the intracellular localization of ataxin-3 as novel treatment approach for Spinocerebellar Ataxia Type 3 (SCA3)” . Congrès IARC 2017, International Ataxia Research Conference 2017, Pise, Italie, 27-30 septembre 2017.