Complete correction of hyperphenylalaninemia following liver-directed, recombinant AAV2/8 vector-mediated gene therapy in murine phenylketonuria.

Harding, C O; Gillingham, M B; Hamman, K; Clark, H; Goebel-Daghighi, E; Bird, A; Koeberl, D D

Harding, C O; Gillingham, M B; Hamman, K; Clark, H; Goebel-Daghighi, E; Bird, A; Koeberl, D D.

Affiliation

Harding CO; Department of Pediatrics, Oregon Health & Science University, Portland, OR 97239, USA. hardingc@ohsu.edu

Gene Ther ; 13(5): 457-62, 2006 Mar.

Article in En | MEDLINE | ID: mdl-16319949

Subject(s)

Dependovirus/genetics; Genetic Therapy/methods; Genetic Vectors/administration & dosage; Liver/metabolism; Phenylalanine Hydroxylase/genetics; Phenylketonurias/therapy; Animals; Female; Gene Expression; Hair Color; Mice; Mice, Inbred C57BL; Models, Animal; Phenylalanine Hydroxylase/blood; Phenylalanine Hydroxylase/metabolism; Phenylketonurias/complications; Phenylketonurias/metabolism; Pigmentation Disorders/etiology; Pigmentation Disorders/metabolism; Pigmentation Disorders/therapy; Recombinant Proteins/administration & dosage; Recombinant Proteins/genetics; Transduction, Genetic/methods

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Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Phenylalanine Hydroxylase / Phenylketonurias / Genetic Therapy / Dependovirus / Genetic Vectors / Liver Type of study: Etiology_studies Limits: Animals Language: En Journal: Gene Ther Year: 2006 Document type: Article

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