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Article

Prenatal enzyme replacement therapy for Akp2−/− mice with lethal hypophosphatasia

5 Jul 2021

Abstract excerpt

Hypophosphatasia (HPP) is a congenital skeletal disease. Impairment of bone mineralization and seizures are due to a deficiency of tissue-nonspecific alkaline phosphatase (TNAP). Enzyme replacement therapy (ERT) is available as a highly successful treatment for pediatric-onset HPP. However, the potential for prenatal ERT has not been fully investigated to date. In this study, we assessed outcomes and maternal...

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PMID: 34277899
DOI: 10.1016/j.reth.2021.06.002

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