Article
iPS-derived neural stem cells for disease modeling and evaluation of therapeutics for mucopolysaccharidosis type II.
Experimental cell research - 1 Mar 2022
Hong Junjie, Cheng Yu-Shan, Yang Shu, Swaroop Manju, Xu Miao, Beers Jeanette, Zou Jizhong, Huang Wenwei, Marugan Juan J, Cai Xiujun, Zheng Wei
Abstract excerpt
Mucopolysaccharidosis type II (MPS II), also known as Hunter syndrome, is a rare, lysosomal disorder caused by mutations in a gene encoding iduronate-2-sulfatase (IDS). IDS deficiency results in an accumulation of glycosaminoglycans (GAGs) and secondary accumulations of other lipids in lysosomes. Symptoms of MPS II include a variety of soft and hard tissue problems, developmental delay, and deterioration of...
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