Successful Gene Therapy in Utero for Lethal Murine Hypophosphatasia

Hypophosphatasia (HPP), caused by mutations in the gene ALPL encoding tissue-nonspecific alkaline phosphatase (TNALP), is an inherited systemic skeletal disease characterized by mineralization defects of bones and teeth. The clinical severity of HPP varies widely, from a lethal perinatal form to mil...

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Bibliografiske detaljer
Main Authors: Sugano, Hanako, Matsumoto, Tae, Miyake, Koichi, Watanabe, Atsushi, Iijima, Osamu, Migita, Makoto, Narisawa, Sonoko, Millán, José Luis, Fukunaga, Yoshitaka, Shimada, Takashi
Format: Artigo
Sprog:Inglês
Udgivet: Mary Ann Liebert, Inc. 2011
Fag:
Research Articles
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC3327603/
https://ncbi.nlm.nih.gov/pubmed/22133046
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/hum.2011.148
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