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Ex Vivo Gene Therapy Treats Bone Complications of Mucopolysaccharidosis Type II Mouse Models through Bone Remodeling Reactivation

Mucopolysaccharidosis type II is a disease caused by organ accumulation of glycosaminoglycans due to iduronate 2-sulfatase deficiency. This study investigated the pathophysiology of the bone complications associated with mucopolysaccharidosis II and the effect of lentivirus-mediated gene therapy of...

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Detalles Bibliográficos
Autores principales:	Wada, Miho, Shimada, Yohta, Iizuka, Sayoko, Ishii, Natsumi, Hiraki, Hiromi, Tachibana, Toshiaki, Maeda, Kazuhiro, Saito, Mitsuru, Arakawa, Shoutaro, Ishimoto, Takuya, Nakano, Takayoshi, Ida, Hiroyuki, Ohashi, Toya, Kobayashi, Hiroshi
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	American Society of Gene & Cell Therapy 2020
Materias:	Original Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7567932/ https://www.ncbi.nlm.nih.gov/pubmed/33102618 http://dx.doi.org/10.1016/j.omtm.2020.09.012

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