ATP7A gene addition to the choroid plexus results in long-term rescue of the lethal copper transport defect in a Menkes disease mouse model

Molecular Therapy

Volumn 19, Issue 12, 2011, Pages 2114-2123

  Donsante, Anthony ^a   Yi, Ling ^a   Zerfas, Patricia M ^a   Brinster, Lauren R ^a   Sullivan, Patricia ^b   Goldstein, David S ^b   Prohaska, Joseph ^c   Centeno, Jose A ^d   Rushing, Elisabeth ^d   Kaler, Stephen G ^a  

^a NATIONAL INSTITUTES OF HEALTH   (United States)

^b NATIONAL INSTITUTE OF NEUROLOGICAL DISORDERS AND STROKE   (United States)

^c UNIVERSITY OF MINNESOTA MEDICAL SCHOOL   (United States)

^d ARMED FORCES INSTITUTE OF PATHOLOGY   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

ADENOSINE TRIPHOSPHATASE; ADENOVIRUS VECTOR; COMPLEMENTARY DNA; COPPER; COPPER EXPORTING ADENOSINE TRIPHOSPHATASE; DOPAMINE; DOPAMINE BETA MONOOXYGENASE; MENKES PROTEIN;

ADENO ASSOCIATED VIRUS; ALLELE; ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; CHOROID PLEXUS; CONTROLLED STUDY; COPPER METABOLISM; FEMALE; GENE MUTATION; GENE THERAPY; LATERAL BRAIN VENTRICLE; LONG TERM CARE; MALE; MENKES SYNDROME; MOUSE; NONHUMAN; PHENOTYPE; RAT; SEQUENCE HOMOLOGY; SEROTYPE; SITE DIRECTED MUTAGENESIS; TREATMENT OUTCOME;

ADENO-ASSOCIATED VIRUS; MURINAE; MUS;

EID: 82955232408     PISSN: 15250016     EISSN: 15250024     Source Type: Journal    
DOI: 10.1038/mt.2011.143     Document Type: Article

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