Title : RNA Interference Therapy for Machado-Joseph Disease: Long-Term Safety Profile of Lentiviral Vectors Encoding Short Hairpin RNAs Targeting Mutant Ataxin-3.

Pub. Date : 2019 Jul

PMID : 30760052






1 Functional Relationships(s)
Download
Sentence
Compound Name
Protein Name
Organism
1 This expansion translates into a long polyglutamine tract, leading to the misfolding of the mutant protein ataxin-3, which abnormally accumulates in the nucleus, thus leading to neurodegeneration in specific brain regions. polyglutamine ataxin 3 Mus musculus