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Article

Prolonged survival and phenotypic correction of Akp2(-/-) hypophosphatasia mice by lentiviral gene therapy.

Journal of bone and mineral research : the official journal of the American Society for Bone and Mineral Research - 1 Jan 2011

Yamamoto Seiko, Orimo Hideo, Matsumoto Tae, Iijima Osamu, Narisawa Sonoko, Maeda Takahide, Millán José Luis, Shimada Takashi

Abstract excerpt

Hypophosphatasia (HPP) is an inherited systemic skeletal disease caused by mutations in the gene encoding the tissue-nonspecific alkaline phosphatase (TNALP) isozyme. The clinical severity of HPP varies widely, with symptoms including rickets and osteomalacia. TNALP knockout (Akp2(-/-)) mice phenotypically mimic the severe infantile form of HPP; that is, TNALP-deficient mice are born with a normal appearance but...

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Identifiers

PMID: 20687159
DOI: 10.1002/jbmr.201
PMCID: PMC3179312

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-08.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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