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

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

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 se...

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Main Authors: Aki Nakamura-Takahashi, Koichi Miyake, Atsushi Watanabe, Yukihiko Hirai, Osamu Iijima, Noriko Miyake, Kumi Adachi, Yuko Nitahara-Kasahara, Hideaki Kinoshita, Taku Noguchi, Shinichi Abe, Sonoko Narisawa, Jose Luis Millán, Takashi Shimada, Takashi Okada
Format: Article
Language:English
Published: Elsevier 2016-01-01
Series:Molecular Therapy: Methods & Clinical Development
Online Access:http://www.sciencedirect.com/science/article/pii/S2329050116301498

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