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

Brain

Volumn 138, Issue 12, 2015, Pages 3555-3566

  Keiser, Megan S ^a   Kordower, Jeffrey H ^b   Gonzalez Alegre, Pedro ^c   Davidson, Beverly L ^c  

^a CHILDREN S HOSPITAL OF PHILADELPHIA   (United States)

^b RUSH MEDICAL COLLEGE   (United States)

^c UNIVERSITY OF PENNSYLVANIA   (United States)

Author keywords

ataxia; gene therapy; neurodegeneration; spinocerebellar ataxia

Indexed keywords

ATAXIN 1; ENHANCED GREEN FLUORESCENT PROTEIN; MESSENGER RNA; MIS1 PROTEIN; UNCLASSIFIED DRUG; VIRUS PROTEIN;

ADENO ASSOCIATED VIRUS 1; ADULT; ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; ASTROCYTOSIS; BRAIN STEM; CELL BODY; CEREBELLUM; CEREBELLUM CORTEX; CEREBELLUM NUCLEUS; CONTROLLED STUDY; DENTATE NUCLEUS; DOWN REGULATION; EMBRYO; ENZYME INHIBITION; ENZYME REPRESSION; EXPERIMENTAL MONKEY; FEMALE; FUNCTIONAL NEUROIMAGING; GENE SILENCING; GENOME; HUMAN; HUMAN CELL; IN VITRO STUDY; INFERIOR OLIVE; MALE; NERVE CELL; NERVE PROJECTION; NERVOUS SYSTEM INFLAMMATION; NONHUMAN; NUCLEAR MAGNETIC RESONANCE IMAGING; PHENOTYPE; PRIORITY JOURNAL; PROTEIN EXPRESSION; PROTEIN LOCALIZATION; PURKINJE CELL; RHESUS MONKEY; RNA INTERFERENCE; SPINOCEREBELLAR DEGENERATION; SPINOCEREBELLAR DEGENERATION TYPE 1; THALAMUS; THALAMUS LATERAL NUCLEUS; VIRAL GENE THERAPY; ANIMAL; DEFICIENCY; DEPENDOPARVOVIRUS; GENE THERAPY; GENETIC TRANSDUCTION; GENETICS; METABOLISM; PROCEDURES; SPINOCEREBELLAR ATAXIAS;

ANIMALS; ATAXIN-1; BRAIN STEM; CEREBELLAR NUCLEI; DEPENDOVIRUS; FEMALE; GENETIC THERAPY; MACACA MULATTA; MALE; PURKINJE CELLS; RNA INTERFERENCE; SPINOCEREBELLAR ATAXIAS; THALAMUS; TRANSDUCTION, GENETIC;

EID: 84951136970     PISSN: 00068950     EISSN: 14602156     Source Type: Journal    
DOI: 10.1093/brain/awv292     Document Type: Article

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