Lentivirus-modified hematopoietic stem cell gene therapy for advanced symptomatic juvenile metachromatic leukodystrophy: a long-term follow-up pilot study

Metachromatic leukodystrophy (MLD) is an inherited disease caused by a deficiency of the enzyme arylsulfatase A (ARSA). Lentivirus-modified autologous hematopoietic stem cell gene therapy (HSCGT) has recently been approved for clinical use in pre and early symptomatic children with MLD to increase A...

Ausführliche Beschreibung

Bibliographische Detailangaben

Veröffentlicht in:	Protein & cell 2025-01, Vol.16 (1), p.16-27
Hauptverfasser:	Zhang, Zhao, Jiang, Hua, Huang, Li, Liu, Sixi, Zhou, Xiaoya, Cai, Yun, Li, Ming, Gao, Fei, Liang, Xiaoting, Tsang, Kam-Sze, Chen, Guangfu, Ma, Chui-Yan, Chai, Yuet-Hung, Liu, Hongsheng, Yang, Chen, Yang, Mo, Zhang, Xiaoling, Han, Shuo, Du, Xin, Chen, Ling, Hwu, Wuh-Liang, Zhuo, Jiacai, Lian, Qizhou
Format:	Artikel
Sprache:	eng
Online-Zugang:	Volltext
Tags:	Tag hinzufügen Keine Tags, Fügen Sie den ersten Tag hinzu!