Gene Therapy Using Recombinant AAV Type 8 Vector Encoding TNAP‐D(10) Improves the Skeletal Phenotypes in Murine Models of Osteomalacia

Hypophosphatasia (HPP), caused by loss‐of‐function mutations in the ALPL gene encoding tissue‐nonspecific alkaline phosphatase (TNAP), is characterized by skeletal and dental hypomineralization that can vary in severity from life‐threatening to milder manifestations only in adulthood. PHOSPHO1 defic...

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Detalles Bibliográficos
Autores principales: Amadeu de Oliveira, Flavia, Mohamed, Fatma F., Kinoshita, Yuka, Narisawa, Sonoko, Farquharson, Colin, Miyake, Koichi, Foster, Brian L, Millan, Jose Luis
Formato: Online Artículo Texto
Lenguaje:English
Publicado: John Wiley & Sons, Inc. 2022
Materias:
Research Articles
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9850441/
https://www.ncbi.nlm.nih.gov/pubmed/36699639
http://dx.doi.org/10.1002/jbm4.10709

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