State-of-the-art 2023 on gene therapy for phenylketonuria.

Martinez, Michael; Harding, Cary O; Schwank, Gerald; Thöny, Beat

Martinez, Michael; Harding, Cary O; Schwank, Gerald; Thöny, Beat.

Afiliação

Martinez M; Department of Molecular and Medical Genetics, Oregon Health & Science University, Portland, Oregon, USA.
Harding CO; Department of Molecular and Medical Genetics, Oregon Health & Science University, Portland, Oregon, USA.
Schwank G; Institute of Pharmacology and Toxicology, University of Zurich, Zurich, Switzerland.
Thöny B; Division of Metabolism, University Children's Hospital Zurich and Children's Research Centre, Zurich, Switzerland.

J Inherit Metab Dis ; 47(1): 80-92, 2024 Jan.

Article em En | MEDLINE | ID: mdl-37401651

Assuntos

Fenilalanina Hidroxilase; Fenilcetonúrias; Humanos; Camundongos; Animais; Fenilalanina Hidroxilase/genética; Fenilcetonúrias/genética; Fenilcetonúrias/terapia; Terapia Genética/métodos; Fígado/patologia; DNA

Palavras-chave

adeno-associated virus; gene editing; gene therapy; mRNA therapy; phenylalanine; phenylalanine hydroxylase; phenylketonuria

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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Fenilalanina Hidroxilase / Fenilcetonúrias Limite: Animals / Humans Idioma: En Revista: J Inherit Metab Dis Ano de publicação: 2024 Tipo de documento: Article País de afiliação: Estados Unidos

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