Transduction of modified factor VIII gene improves lentiviral gene therapy efficacy for hemophilia A.

Gong, Jie; Chung, Tsai-Hua; Zheng, Jie; Zheng, Huyong; Chang, Lung-Ji

Gong, Jie; Chung, Tsai-Hua; Zheng, Jie; Zheng, Huyong; Chang, Lung-Ji.

Afiliación

Gong J; School of Medicine, University of Electronic Science and Technology of China, Sichuan, China.
Chung TH; School of Medicine, University of Electronic Science and Technology of China, Sichuan, China; Shenzhen Geno-Immune Medical Institute, Shenzhen, China.
Zheng J; Hematology Center, Beijing Children's Hospital, Capital Medical University, Beijing, China.
Zheng H; Hematology Center, Beijing Children's Hospital, Capital Medical University, Beijing, China.
Chang LJ; School of Medicine, University of Electronic Science and Technology of China, Sichuan, China; Shenzhen Geno-Immune Medical Institute, Shenzhen, China; Hematology Center, Beijing Children's Hospital, Capital Medical University, Beijing, China. Electronic address: c@szgimi.org.

J Biol Chem ; 297(6): 101397, 2021 12.

Article en En | MEDLINE | ID: mdl-34774524

Asunto(s)

Factor VIII; Terapia Genética; Vectores Genéticos; Hemofilia A; Lentivirus; Transducción Genética; Factor VIII/biosíntesis; Factor VIII/genética; Hemofilia A/genética; Hemofilia A/metabolismo; Hemofilia A/terapia; Humanos; Células K562

Palabras clave

FVIII; gene therapy; glycosylation; hemophilia A; lentiviral vector

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Texto completo: 1 Colección: 01-internacional Banco de datos: MEDLINE Asunto principal: Transducción Genética / Factor VIII / Terapia Genética / Lentivirus / Vectores Genéticos / Hemofilia A Límite: Humans Idioma: En Revista: J Biol Chem Año: 2021 Tipo del documento: Article País de afiliación: China

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