Article

Successful Gene Therapy <i>in Utero</i> for Lethal Murine Hypophosphatasia

2011-12-02

Abstract excerpt

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 mild odontohypophosphatasia showing only dental manifestations. HPP model mice (Akp2−/−) phenotypically mimic the severe...

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Literature Corpus work: 64d253a6-a77f-5a2f-a9dc-a1fb957e3501
DOI: 10.1089/hum.2011.148

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