Induced Pluripotent Stem Cell Derivation and Ex Vivo Gene Correction Using a Mucopolysaccharidosis Type 1 Disease Mouse Model

• Hauptverfasser: Toshio Miki (Verfasst von), Ludivina Vazquez (Verfasst von), Lisa Yanuaria (Verfasst von), Omar Lopez (Verfasst von), Irving M. Garcia (Verfasst von), Kazuo Ohashi (Verfasst von), Natalie S. Rodriguez (Verfasst von)
• Format: Buch
• Veröffentlicht: Hindawi Limited, 2019-01-01T00:00:00Z.
• Schlagworte: article
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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...