Reducing the transcriptional read-through rate of a lentiviral vector for ß-thalassemia gene therapy.

Wu, Jiahui; Chen, Yuan; Shen, Wenchen; Zhang, Jingzhi; Zeng, Fanyi

Wu, Jiahui; Chen, Yuan; Shen, Wenchen; Zhang, Jingzhi; Zeng, Fanyi.

Afiliación

Wu J; Shanghai Institute of Medical Genetics, Shanghai Children's Hospital, Shanghai Jiao Tong University School of Medicine, Shanghai, China.
Chen Y; NHC Key Laboratory of Medical Embryogenesis and Developmental Molecular Biology & Shanghai Key Laboratory of Embryo and Reproduction Engineering, Shanghai, China.
Shen W; Shanghai Institute of Medical Genetics, Shanghai Children's Hospital, Shanghai Jiao Tong University School of Medicine, Shanghai, China.
Zhang J; NHC Key Laboratory of Medical Embryogenesis and Developmental Molecular Biology & Shanghai Key Laboratory of Embryo and Reproduction Engineering, Shanghai, China.
Zeng F; Shanghai Institute of Medical Genetics, Shanghai Children's Hospital, Shanghai Jiao Tong University School of Medicine, Shanghai, China.

J Gene Med ; 26(1): e3640, 2024 Jan.

Article en En | MEDLINE | ID: mdl-37989259

Asunto(s)

Talasemia beta; Humanos; Talasemia beta/genética; Talasemia beta/terapia; Lentivirus/genética; Vectores Genéticos/genética; Terapia Genética/métodos; Globinas beta/genética

Palabras clave

C-U+; EATRT; WPRE; lentiviral vector; pro-BB305; ß-thalassemia

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Texto completo: 1 Bases de datos: MEDLINE Asunto principal: Talasemia beta Límite: Humans Idioma: En Revista: J Gene Med Asunto de la revista: BIOLOGIA MOLECULAR / GENETICA MEDICA Año: 2024 Tipo del documento: Article País de afiliación: China

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