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...

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Pubblicato in:Mol Ther Methods Clin Dev
Autori principali: Nakamura-Takahashi, Aki, Miyake, Koichi, Watanabe, Atsushi, Hirai, Yukihiko, Iijima, Osamu, Miyake, Noriko, Adachi, Kumi, Nitahara-Kasahara, Yuko, Kinoshita, Hideaki, Noguchi, Taku, Abe, Shinichi, Narisawa, Sonoko, Millán, Jose Luis, Shimada, Takashi, Okada, Takashi
Natura: Artigo
Lingua:Inglês
Pubblicazione: Nature Publishing Group 2016
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Article
Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC4739158/
https://ncbi.nlm.nih.gov/pubmed/26904710
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mtm.2015.59
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