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

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...

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Библиографические подробности
Опубликовано в: :Hum Gene Ther
Главные авторы: 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
Формат: Artigo
Язык:Inglês
Опубликовано: Mary Ann Liebert, Inc. 2015
Предметы:
Research Articles
Online-ссылка:https://ncbi.nlm.nih.gov/pmc/articles/PMC5248541/
https://ncbi.nlm.nih.gov/pubmed/26467745
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/hum.2015.078
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