Pre-clinical efficacy and dosing of an AAV8 vector expressing human methylmalonyl-CoA mutase in a murine model of methylmalonic acidemia (MMA)

Molecular Genetics and Metabolism

Volumn 107, Issue 3, 2012, Pages 617-619

  Chandler, Randy J ^a   Venditti, Charles P ^a  

^a NATIONAL HUMAN GENOME RESEARCH INSTITUTE   (United States)

Author keywords

AAV; Adeno associated virus; Gene therapy; Methylmalonic acidemia; MMA; Organic acidemia

Indexed keywords

ADENO ASSOCIATED VIRUS 8 VECTOR; COMPLEMENTARY DNA; METHYLMALONYL COENZYME A MUTASE; PARVOVIRUS VECTOR; UNCLASSIFIED DRUG;

ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; CONTROLLED STUDY; DRUG EFFICACY; GENE DOSAGE; GENE THERAPY; HUMAN; HUMAN TISSUE; IMMUNE RESPONSE; METHYLMALONIC ACIDEMIA; MITOCHONDRION; MOUSE; NONHUMAN; PHENOTYPE; PRIORITY JOURNAL;

AMINO ACID METABOLISM, INBORN ERRORS; AMINO ACID SEQUENCE; ANIMALS; DEPENDOVIRUS; DISEASE MODELS, ANIMAL; GENE TRANSFER TECHNIQUES; GENETIC THERAPY; GENETIC VECTORS; HUMANS; METHYLMALONYL-COA MUTASE; MICE; MICE, KNOCKOUT; MOLECULAR SEQUENCE DATA; PHENOTYPE; SEQUENCE ALIGNMENT; SURVIVAL RATE;

ADENO-ASSOCIATED VIRUS; MURINAE; MUS;

EID: 84867897305     PISSN: 10967192     EISSN: 10967206     Source Type: Journal    
DOI: 10.1016/j.ymgme.2012.09.019     Document Type: Article

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