RNA Interference Mitigates Motor and Neuropathological Deficits in a Cerebellar Mouse Model of Machado-Joseph Disease

Toward RNAi Therapy for the Polyglutamine Disease Machado–Joseph Disease

(2013) Maria do Carmo Costa et al.   MOLECULAR THERAPY

Silencing Mutant ATXN3 Expression Resolves Molecular Phenotypes in SCA3 Transgenic Mice

(2013) Edgardo Rodríguez-Lebrón et al.   MOLECULAR THERAPY

Silencing Mutant Ataxin-3 Rescues Motor Deficits and Neuropathology in Machado-Joseph Disease Transgenic Mice

(2013) Clévio Nóbrega et al.   PLoS One

Calpastatin-mediated inhibition of calpains in the mouse brain prevents mutant ataxin 3 proteolysis, nuclear localization and aggregation, relieving Machado-Joseph disease

(2012) A. T. Simoes et al.   BRAIN

Overexpression of the autophagic beclin-1 protein clears mutant ataxin-3 and alleviates Machado–Joseph disease

(2011) Isabel Nascimento-Ferreira et al.   BRAIN

Absence of ataxin-3 leads to cytoskeletal disorganization and increased cell death

(2010) Ana-João Rodrigues et al.   BIOCHIMICA ET BIOPHYSICA ACTA-MOLECULAR CELL RESEARCH

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

(2010) Sandro Alves et al.   HUMAN MOLECULAR GENETICS

Mouse Ataxin-3 Functional Knock-Out Model

(2010) Pawel M. Switonski et al.   NEUROMOLECULAR MEDICINE

Sustained effects of nonallele-specificHuntingtinsilencing

(2009) Valérie Drouet et al.   ANNALS OF NEUROLOGY

Reduced exploration, increased anxiety, and altered social behavior: Autistic-like features of euchromatin histone methyltransferase 1 heterozygous knockout mice

(2009) Monique C.M. Balemans et al.   BEHAVIOURAL BRAIN RESEARCH

Allele-specific RNAi Mitigates Phenotypic Progression in a Transgenic Model of Alzheimer's Disease

(2009) Edgardo Rodríguez-Lebrón et al.   MOLECULAR THERAPY

A transgenic mouse model of spinocerebellar ataxia type 3 resembling late disease onset and gender-specific instability of CAG repeats

(2009) Jana Boy et al.   NEUROBIOLOGY OF DISEASE

Caring for Machado–Joseph disease: Current understanding and how to help patients

(2009) Anelyssa D'Abreu et al.   PARKINSONISM & RELATED DISORDERS

Sleep symptoms and their clinical correlates in Machado-Joseph disease

(2008) A. D’Abreu et al.   ACTA NEUROLOGICA SCANDINAVICA

Lentivector-mediated rescue from cerebellar ataxia in a mouse model of spinocerebellar ataxia

(2008) Takashi Torashima et al.   EMBO REPORTS

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

(2008) Sandro Alves et al.   HUMAN MOLECULAR GENETICS

Early symptoms in spinocerebellar ataxia type 1, 2, 3, and 6

(2008) Christoph Globas et al.   MOVEMENT DISORDERS

Polyglutamine-expanded ataxin-3 causes cerebellar dysfunction of SCA3 transgenic mice by inducing transcriptional dysregulation

(2008) An-Hsun Chou et al.   NEUROBIOLOGY OF DISEASE

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