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

• Main Authors: Toshio Miki (Author), Ludivina Vazquez (Author), Lisa Yanuaria (Author), Omar Lopez (Author), Irving M. Garcia (Author), Kazuo Ohashi (Author), Natalie S. Rodriguez (Author)
• Format: Book
• Published: Hindawi Limited, 2019-01-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

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...