Gene therapy for aromatic L-amino acid decarboxylase deficiency

Science Translational Medicine

Volumn 4, Issue 134, 2012, Pages

  Hwu, Wuh Liang ^a,b   Muramatsu, Shin Ichi ^c   Tseng, Sheng Hong ^a   Tzen, Kai Yuan ^a,b   Lee, Ni Chung ^a   Chien, Yin Hsiu ^a   Snyder, Richard O ^d   Byrne, Barry J ^d   Tai, Chun Hwei ^a   Wu, Ruey Meei ^a  

^a NATIONAL TAIWAN UNIVERSITY HOSPITAL   (Taiwan)

^b NATIONAL TAIWAN UNIVERSITY   (Taiwan)

^c JICHI MEDICAL UNIVERSITY   (Japan)

^d UNIVERSITY OF FLORIDA COLLEGE OF MEDICINE   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

6 FLUORODOPA F 18; AROMATIC LEVO AMINO ACID DECARBOXYLASE; PARVOVIRUS VECTOR;

AADC GENE; ADOLESCENT; APNEA; AROMATIC LEVO AMINO ACID DECARBOXYLASE DEFICIENCY; ARTICLE; BRAIN FUNCTION; BRAIN REGION; CEREBROSPINAL FLUID ANALYSIS; CHILD; CHILD DEVELOPMENT; CLINICAL ARTICLE; CLINICAL EVALUATION; COGNITION; CYANOSIS; DRUG EFFICACY; ENZYME DEFICIENCY; FEMALE; GENE; GENE MUTATION; GENE THERAPY; HUMAN; MALE; MOTOR PERFORMANCE; OROFACIAL DYSKINESIA; POSITRON EMISSION TOMOGRAPHY; PRIORITY JOURNAL; PUTAMEN; SCHOOL CHILD; TREATMENT RESPONSE; VIRAL GENE DELIVERY SYSTEM;

AMINO ACID METABOLISM, INBORN ERRORS; ANTIBODIES; AROMATIC-L-AMINO-ACID DECARBOXYLASES; CHILD; CHILD, PRESCHOOL; DEMOGRAPHY; DEPENDOVIRUS; DIHYDROXYPHENYLALANINE; FEMALE; GENE TRANSFER TECHNIQUES; GENETIC THERAPY; GENETIC VECTORS; HUMANS; MALE; MOTOR ACTIVITY; NEUROTRANSMITTER AGENTS; POSITRON-EMISSION TOMOGRAPHY; TAIWAN;

EID: 84861139370     PISSN: 19466234     EISSN: 19466242     Source Type: Journal    
DOI: 10.1126/scitranslmed.3003640     Document Type: Article

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