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Stem Cell Therapy: Cell‐Based Therapy for Canavan Disease Using Human iPSC‐Derived NPCs and OPCs (Adv. Sci. 23/2020)
Advanced Science ( IF 15.1 ) Pub Date : 2020-12-02 , DOI: 10.1002/advs.202070131
Lizhao Feng , Jianfei Chao , E Tian , Li Li , Peng Ye , Mi Zhang , Xianwei Chen , Qi Cui , Guihua Sun , Tao Zhou , Gerardo Felix , Yue Qin , Wendong Li , Edward David Meza , Jeremy Klein , Lucy Ghoda , Weidong Hu , Yonglun Luo , Wei Dang , David Hsu , Joseph Gold , Steven A. Goldman , Reuben Matalon , Yanhong Shi

Canavan disease (CD) is a leukodystrophy caused by aspartoacylase (ASPA) gene mutation with no cure so far. In this study, a functional ASPA was introduced into patient iPSC‐derived neural progenitor cells (iNPCs) and the engineered ASPA iNPCs could rescue major pathological features of CD in a disease mouse model. These patient iPSC‐derived cellular products are promising cell therapies for CD patients. [Image designed and prepared by Olivia Sun]
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中文翻译:

干细胞疗法:使用人类iPSC衍生的NPC和OPC进行基于细胞的Canavan疾病疗法(Adv。Sci。23/2020)

Canavan病(CD)是由天冬氨酸酰化酶(ASPA)基因突变引起的白细胞营养不良,目前尚无治愈方法。在这项研究中,功能性ASPA被引入患者iPSC衍生的神经祖细胞(iNPC)中,工程化的ASPA iNPC可以挽救疾病小鼠模型中CD的主要病理特征。这些患者iPSC衍生的细胞产品是CD患者的有前途的细胞疗法。[图片由Olivia Sun设计和制作]
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更新日期:2020-12-03
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