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...

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Detalles Bibliográficos
Autores principales: Miki, Toshio, Vazquez, Ludivina, Yanuaria, Lisa, Lopez, Omar, Garcia, Irving M., Ohashi, Kazuo, Rodriguez, Natalie S.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Hindawi 2019
Materias:
Research Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6466856/
https://www.ncbi.nlm.nih.gov/pubmed/31065277
http://dx.doi.org/10.1155/2019/6978303

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6466856/
https://www.ncbi.nlm.nih.gov/pubmed/31065277
http://dx.doi.org/10.1155/2019/6978303

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