Lack of toxicity of alpha-sarcoglycan overexpression supports clinical gene transfer trial in LGMD2D

Neurology

Volumn 71, Issue 4, 2008, Pages 240-247

  Rodino Klapac, L R ^a   Lee, J S ^b   Mulligan, R C ^a   Clark, K R ^b   Mendell, J R ^a,c  

^a OHIO STATE UNIVERSITY   (United States)

^b HARVARD MEDICAL SCHOOL   (United States)

^c O   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

ADENOVIRUS VECTOR; ALPHA SARCOGLYCAN; CREATINE KINASE MM; DESMIN; CREATINE KINASE; SARCOGLYCAN;

ADENOVIRUS; ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; CELL STRAIN HEK293; CONTROLLED STUDY; CYTOMEGALOVIRUS; DRUG DOSE ESCALATION; DRUG DOSE REDUCTION; DRUG MEGADOSE; GENE EXPRESSION; GENE OVEREXPRESSION; GENE TRANSFER; HUMAN; HUMAN CELL; KNOCKOUT MOUSE; LIMB GIRDLE MUSCULAR DYSTROPHY; LOW DRUG DOSE; MOUSE; MUSCLE CELL; NONHUMAN; PRIORITY JOURNAL; PROMOTER REGION; TIBIALIS ANTERIOR MUSCLE; VIRAL GENE THERAPY; WESTERN BLOTTING; ADENO ASSOCIATED VIRUS; ANIMAL; BIOSYNTHESIS; C57BL MOUSE; CLINICAL TRIAL; GENE THERAPY; GENE VECTOR; GENETICS; IMMUNOLOGY; METABOLISM; METHODOLOGY; MOUSE MUTANT; MYOSITIS; PATHOPHYSIOLOGY; SKELETAL MUSCLE; STANDARD; TREATMENT OUTCOME; UPREGULATION;

ANIMALS; CLINICAL TRIALS AS TOPIC; CREATINE KINASE; CYTOMEGALOVIRUS; DEPENDOVIRUS; GENE EXPRESSION; GENE THERAPY; GENE TRANSFER TECHNIQUES; GENETIC VECTORS; HUMANS; MICE; MICE, INBRED C57BL; MICE, KNOCKOUT; MUSCLE, SKELETAL; MUSCULAR DYSTROPHIES, LIMB-GIRDLE; MYOSITIS; PROMOTER REGIONS (GENETICS); SARCOGLYCANS; TREATMENT OUTCOME; UP-REGULATION;

EID: 50549085053     PISSN: 00283878     EISSN: 1526632X     Source Type: Journal    
DOI: 10.1212/01.wnl.0000306309.85301.e2     Document Type: Article

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