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Article

Improvement of skeletal lesions in mice with mucopolysaccharidosis type VII by neonatal adenoviral gene transfer.

Molecular therapy : the journal of the American Society of Gene Therapy - 1 Nov 2003

Kanaji Arihiko, Kosuga Motomichi, Li Xiao Kang, Fukuhara Yasuyuki, Tanabe Akiko, Kamata Yuko, Azuma Noriyuki, Yamada Masao, Sakamaki Toyonori, Toyama Yoshiaki, Okuyama Torayuki

Abstract excerpt

Neonatal gene transfer using adenovirus vectors expressing human beta-glucuronidase (AxCAhGUS) resulted in pathological improvement in multiple visceral organs of mice with mucopolysaccharidosis type VII (MPSVII). However, the therapeutic effect on skeletal deformities and growth retardation, the major clinical symptoms in MPSVII, was not fully investigated by biochemical and histopathological analyses. In this...

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Source

Identifiers

PMID: 14599804
DOI: 10.1016/j.ymthe.2003.07.004

Genes

GUSB

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-08.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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