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Article

Prevention of Lethal Murine Hypophosphatasia by Neonatal Ex Vivo Gene Therapy Using Lentivirally Transduced Bone Marrow Cells.

Human gene therapy - 1 Dec 2015

Iijima Osamu, Miyake Koichi, Watanabe Atsushi, Miyake Noriko, Igarashi Tsutomu, Kanokoda Chizu, Nakamura-Takahashi Aki, Kinoshita Hideaki, Noguchi Taku, Abe Shinichi, Narisawa Sonoko, Millán José Luis, Okada Takashi, Shimada Takashi

Abstract excerpt

Hypophosphatasia (HPP) is an inherited skeletal and dental disease caused by loss-of-function mutations in the gene that encodes tissue-nonspecific alkaline phosphatase (TNALP). The major symptoms of severe forms of the disease are bone defects, respiratory insufficiency, and epileptic seizures. In 2015, enzyme replacement therapy (ERT) using recombinant bone-targeted TNALP with deca-aspartate (D10) motif was...

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Identifiers

PMID: 26467745
DOI: 10.1089/hum.2015.078
PMCID: PMC5248541

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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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