Prenatal enzyme replacement therapy for Akp2 ^-/- mice with lethal hypophosphatasia.

ABSTRACT: 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 safety using a combinational approach with prenatal and postnatal administration of recombinant TNAP in Akp2 ^-/- mice as a model of infantile HPP. For the prenatal ERT, we administered subcutaneous injections of recombinant TNAP to pregnant mice from embryonic day 11.5-14.5 until delivery, and then sequentially to Akp2 ^-/- pups from bi

SUBMITTER: Hasegawa A 

PROVIDER: S-EPMC8267436 | biostudies-literature | 2021 Dec

REPOSITORIES: biostudies-literature