Allele-specific RNAi selectively silences mutant SOD1 and achieves significant therapeutic benefit in vivo

Neurobiology of Disease

Volumn 23, Issue 3, 2006, Pages 578-586

  Xia, Xugang ^a   Zhou, Hongxia ^a   Huang, Yong ^a   Xu, Zuoshang ^a  

^a UNIVERSITY OF MASSACHUSETTS MEDICAL SCHOOL   (United States)

Author keywords

Cre lox; Dominant; Gain of function; Inducible; Motor neuron; Neurodegeneration; Neurodegenerative disease; Regulatable; RNAi therapy; Transgenic mice

Indexed keywords

COPPER ZINC SUPEROXIDE DISMUTASE; SHORT HAIRPIN RNA;

ALLELE; AMYOTROPHIC LATERAL SCLEROSIS; ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL TISSUE; ARTICLE; CONTROLLED STUDY; EMBRYO; GENE EXPRESSION; GENE FUNCTION; GENE SILENCING; HUMAN; HUMAN CELL; IN VIVO STUDY; MOUSE; NONHUMAN; PRIORITY JOURNAL; RNA INTERFERENCE; STATISTICAL SIGNIFICANCE; SURVIVAL TIME; TRANSGENIC MOUSE; WILD TYPE;

ALLELES; AMYOTROPHIC LATERAL SCLEROSIS; ANIMALS; CENTRAL NERVOUS SYSTEM; DISEASE MODELS, ANIMAL; DOWN-REGULATION; GENE SILENCING; GENE THERAPY; HUMANS; MICE; MICE, INBRED C57BL; MICE, TRANSGENIC; MOTOR NEURONS; MUTATION; RNA INTERFERENCE; RNA, SMALL INTERFERING; SUPEROXIDE DISMUTASE; SURVIVAL RATE; TREATMENT OUTCOME;

EID: 33747201641     PISSN: 09699961     EISSN: None     Source Type: Journal    
DOI: 10.1016/j.nbd.2006.04.019     Document Type: Article

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