Treatment of hypophosphatasia by muscle-directed expression of bone-targeted alkaline phosphatase via self-complementary AAV8 vector

Abstract

Hypophosphatasia (HPP) is an inherited disease caused by genetic mutations in the gene encoding tissue-nonspecific alkaline phosphatase (TNALP). This results in defects in bone and tooth mineralization. We recently demonstrated that TNALP-deficient (Akp2 (-/-) ) mice, which mimic the phenotype of the severe infantile form of HPP, can be treated by… (More)
DOI: 10.1038/mtm.2015.59

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@inproceedings{NakamuraTakahashi2016TreatmentOH, title={Treatment of hypophosphatasia by muscle-directed expression of bone-targeted alkaline phosphatase via self-complementary AAV8 vector}, author={Aki Nakamura-Takahashi and Koichi Miyake and Atsushi Watanabe and Yukihiko Hirai and Osamu T Iijima and Noriko Miyake and Kumi Adachi and Yuko Nitahara-Kasahara and Hideaki Kinoshita and Taku Noguchi and Shin-Ichi Abe and Sonoko Narisawa and Jos{\'e} Luis Mill{\'a}n and Takashi L Shimada and Takashi Okada}, booktitle={Molecular therapy. Methods & clinical development}, year={2016} }