Sustained enzymatic correction by rAAV-mediated liver gene therapy protects against induced motor neuropathy in acute porphyria mice

Molecular Therapy

Volumn 19, Issue 2, 2011, Pages 243-250

  Unzu, Carmen ^a   Sampedro, Ana ^a   Mauleón, Itsaso ^a   Alegre, Manuel ^b   Beattie, Stuart G ^c   De Salamanca, Rafael Enríquez ^d   Snapper, Jolanda ^c   Twisk, Jaap ^c   Petry, Harald ^c   González Aseguinolaza, Gloria ^a   Artieda, Julio ^b   Rodríguez Pena, María Sol ^c   Prieto, Jess ^a,e   Fontanellas, Antonio ^a  

^a UNIVERSITY OF NAVARRA   (Spain)

^b UNIVERSITY CLINIC OF NAVARRA   (Spain)

^c Research and Development   (Netherlands)

^d HOSPITAL UNIVERSITARIO 12 DE OCTUBRE   (Spain)

^e INSTITUTO DE SALUD CARLOS III   (Spain)

Author keywords

[No Author keywords available]

Indexed keywords

ALANINE AMINOTRANSFERASE; PARVOVIRUS VECTOR; PORPHOBILINOGEN DEAMINASE;

ACUTE INTERMITTENT PORPHYRIA; ANIMAL EXPERIMENT; ANIMAL MODEL; ARTICLE; CONTROLLED STUDY; DRUG EFFICACY; DRUG MEGADOSE; DRUG SAFETY; FEMALE; GENE EXPRESSION; GENETIC TRANSDUCTION; GENETIC VARIABILITY; LIVER CELL; LIVER FUNCTION TEST; MALE; MOTOR NEUROPATHY; MOUSE; NERVE FIBER; NERVE FIBER DEGENERATION; NONHUMAN; PROTEIN URINE LEVEL; SCIATIC NERVE; SEROTYPE; TREATMENT RESPONSE; VIRAL GENE THERAPY;

ADENO-ASSOCIATED VIRUS; ANIMALIA; MUS;

EID: 85027918045     PISSN: 15250016     EISSN: 15250024     Source Type: Journal    
DOI: 10.1038/mt.2010.210     Document Type: Article

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