Adeno-associated virus-mediated gene therapy for rare pediatric neurogenetic diseases: Current status and outlook.

Wang, Xiaole; Lin, Xueqin; He, Hailan; Peng, Jing

Adeno-associated virus-mediated gene therapy for rare pediatric neurogenetic diseases: Current status and outlook. / èºç¸å³çæ¯ä»å¯¼çåºå æ²»çå¨å¿ç«¥ç¥ç»éä¼ ç½è§çä¸åºç¨çç°ç¶ä¸å±æ.

Wang, Xiaole; Lin, Xueqin; He, Hailan; Peng, Jing.

Afiliación

Wang X; Department of Pediatrics, Xiangya Hospital, Central South University, Changsha 410008. wangxl315@csu.edu.cn.
Lin X; Department of Pediatrics, Xiangya Hospital, Central South University, Changsha 410008.
He H; Department of Pediatrics, Xiangya Hospital, Central South University, Changsha 410008.
Peng J; Department of Pediatrics, Xiangya Hospital, Central South University, Changsha 410008. pengjing627@126.com.

Zhong Nan Da Xue Xue Bao Yi Xue Ban ; 48(9): 1388-1396, 2023.

Article en En, Zh | MEDLINE | ID: mdl-38044650

Asunto(s)

Dependovirus; Distrofia Muscular de Duchenne; Estados Unidos; Niño; Humanos; Dependovirus/genética; Enfermedades Raras/genética; Enfermedades Raras/terapia; Distrofia Muscular de Duchenne/genética; Distrofia Muscular de Duchenne/terapia; Terapia Genética; Vectores Genéticos

Palabras clave

adeno-associated virus; gene therapy; rare pediatric neurogenetic diseases

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Texto completo: 1 Base de datos: MEDLINE Asunto principal: Dependovirus / Distrofia Muscular de Duchenne País/Región como asunto: America do norte Idioma: En / Zh Revista: Zhong Nan Da Xue Xue Bao Yi Xue Ban Asunto de la revista: MEDICINA Año: 2023 Tipo del documento: Article

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