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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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Detalles Bibliográficos
Autores principales:	Matsumoto, Tae, Miyake, Koichi, Miyake, Noriko, Iijima, Osamu, Adachi, Kumi, Narisawa, Sonoko, Millán, José Luis, Orimo, Hideo, Shimada, Takashi
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	American Society of Gene & Cell Therapy 2021
Materias:	Original Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8408425/ https://www.ncbi.nlm.nih.gov/pubmed/34514025 http://dx.doi.org/10.1016/j.omtm.2021.06.006

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