RNAi prevents and reverses phenotypes induced by mutant human ataxin-1

Broad distribution of ataxin 1 silencing in rhesus cerebella for spinocerebellar ataxia type 1 therapy

(2015) Megan S. Keiser et al.   BRAIN

In vivo neurometabolic profiling in patients with spinocerebellar ataxia types 1, 2, 3, and 7

(2015) Isaac M. Adanyeguh et al.   MOVEMENT DISORDERS

Early activation of microglia and astrocytes in mouse models of spinocerebellar ataxia type 1

(2015) M. Cvetanovic et al.   NEUROSCIENCE

Broad Therapeutic Benefit After RNAi Expression Vector Delivery to Deep Cerebellar Nuclei: Implications for Spinocerebellar Ataxia Type 1 Therapy

(2014) Megan S Keiser et al.   MOLECULAR THERAPY

Single nucleotide seed modification restores in vivo tolerability of a toxic artificial miRNA sequence in the mouse brain

(2014) Alex Mas Monteys et al.   NUCLEIC ACIDS RESEARCH

RNAi or overexpression: Alternative therapies for Spinocerebellar Ataxia Type 1

(2013) Megan S. Keiser et al.   NEUROBIOLOGY OF DISEASE

Altered Purkinje cell miRNA expression and SCA1 pathogenesis

(2013) Edgardo Rodriguez-Lebron et al.   NEUROBIOLOGY OF DISEASE

In vivo monitoring of recovery from neurodegeneration in conditional transgenic SCA1 mice

(2011) Gülin Öz et al.   EXPERIMENTAL NEUROLOGY

Noninvasive Detection of Presymptomatic and Progressive Neurodegeneration in a Mouse Model of Spinocerebellar Ataxia Type 1

(2010) G. Oz et al.   JOURNAL OF NEUROSCIENCE

Neurochemical alterations in spinocerebellar ataxia type 1 and their correlations with clinical status

(2010) Gülin Öz et al.   MOVEMENT DISORDERS