Gene therapy for inherited metabolic diseases.

Yilmaz, Berna Seker; Gurung, Sonam; Perocheau, Dany; Counsell, John; Baruteau, Julien

Yilmaz, Berna Seker; Gurung, Sonam; Perocheau, Dany; Counsell, John; Baruteau, Julien.

Afiliación

Yilmaz BS; Genetics and Genomic Medicine, Great Ormond Street Institute of Child Health, University College London, London, UK.
Gurung S; Department of Paediatric Metabolic Medicine, Faculty of Medicine, Mersin University, Mersin, Turkey.
Perocheau D; Genetics and Genomic Medicine, Great Ormond Street Institute of Child Health, University College London, London, UK.
Counsell J; Genetics and Genomic Medicine, Great Ormond Street Institute of Child Health, University College London, London, UK.
Baruteau J; Developmental Neurosciences Research and Teaching Department, Great Ormond Street Institute of Child Health, University College London, London, UK.

J Mother Child ; 24(2): 53-64, 2020 Nov 10.

Article en En | MEDLINE | ID: mdl-33554501

Asunto(s)

Técnicas de Transferencia de Gen; Terapia Genética/métodos; Enfermedades Metabólicas/genética; Enfermedades Metabólicas/terapia; Enfermedades Raras/genética; Enfermedades Raras/terapia; Humanos

Palabras clave

adeno-associated virus; gene therapy; inherited metabolic disease; lentivirus; messenger RNA; zinc finger nuclease

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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Terapia Genética / Técnicas de Transferencia de Gen / Enfermedades Raras / Enfermedades Metabólicas Límite: Humans Idioma: En Revista: J Mother Child Año: 2020 Tipo del documento: Article País de afiliación: Reino Unido Pais de publicación: Polonia

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