Treatment with bone maturation and average lifespan of HPP model mice by AAV8-mediated neonatal gene therapy via single muscle injection

Treatment with bone maturation and average lifespan of HPP model mice by AAV8-mediated neonatal gene therapy via single muscle injection

Hypophosphatasia (HPP) is an inherited skeletal disease characterized by defective bone and tooth mineralization due to a deficiency in tissue-nonspecific alkaline phosphatase (TNALP). Patients with the severe infantile form of HPP may appear normal at birth, but their prognosis is very poor. To dev...

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Bibliographic Details
Main Authors: Tae Matsumoto, Koichi Miyake, Noriko Miyake, Osamu Iijima, Kumi Adachi, Sonoko Narisawa, José Luis Millán, Hideo Orimo, Takashi Shimada
Format: Article
Language:English
Published: Elsevier 2021-09-01
Series:Molecular Therapy: Methods & Clinical Development
Subjects:
hypophosphatasia
adeno-associated virus vector
neonatal gene therapy
muscle injection
tissue-nonspecific alkaline phosphatase
Online Access:http://www.sciencedirect.com/science/article/pii/S232905012100108X

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http://www.sciencedirect.com/science/article/pii/S232905012100108X

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