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Treatment of hypophosphatasia by muscle-directed expression of bone-targeted alkaline phosphatase via self-complementary AAV8 vector.
Nakamura-Takahashi A, Miyake K, Watanabe A, Hirai Y, Iijima O, Miyake N, Adachi K, Nitahara-Kasahara Y, Kinoshita H, Noguchi T, Abe S, Narisawa S, Millán JL, Shimada T, Okada T. Nakamura-Takahashi A, et al. Mol Ther Methods Clin Dev. 2016 Feb 3;3:15059. doi: 10.1038/mtm.2015.59. eCollection 2016. Mol Ther Methods Clin Dev. 2016. PMID: 26904710 Free PMC article.
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