Silencing ataxin-3 mitigates degeneration in a rat model of Machado–Joseph disease: no role for wild-type ataxin-3?

Dopamine Gene Therapy for Parkinson's Disease in a Nonhuman Primate Without Associated Dyskinesia

(2009) B. Jarraya et al.   Science Translational Medicine

Identification and Allele-Specific Silencing of the Mutant Huntingtin Allele in Huntington's Disease Patient-Derived Fibroblasts

(2008) P.H.J. van Bilsen et al.   HUMAN GENE THERAPY

Striatal and nigral pathology in a lentiviral rat model of Machado-Joseph disease

(2008) Sandro Alves et al.   HUMAN MOLECULAR GENETICS

The Deubiquitinating Enzyme Ataxin-3, a Polyglutamine Disease Protein, Edits Lys63Linkages in Mixed Linkage Ubiquitin Chains

(2008) Brett J. Winborn et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

AAV Vector–mediated RNAi of Mutant Huntingtin Expression Is Neuroprotective in a Novel Genetic Rat Model of Huntington's Disease

(2008) Nicholas R Franich et al.   MOLECULAR THERAPY

Allele-Specific RNA Silencing of Mutant Ataxin-3 Mediates Neuroprotection in a Rat Model of Machado-Joseph Disease

(2008) Sandro Alves et al.   PLoS One