Induced Pluripotent Stem Cell Derivation and Ex Vivo Gene Correction Using a Mucopolysaccharidosis Type 1 Disease Mouse Model
Mucopolysaccharidosis type 1 (MPS-1), also known as Hurler’s disease, is a congenital metabolic disorder caused by a mutation in the alpha-L-iduronidase (IDUA) gene, which results in the loss of lysosomal enzyme function for the degradation of glycosaminoglycans. Here, we demonstrate the proof of co...
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Main Authors: | , , , , , , |
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Format: | Article |
Language: | English |
Published: |
Wiley
2019-01-01
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Series: | Stem Cells International |
Online Access: | http://dx.doi.org/10.1155/2019/6978303 |
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