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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...
Autores principales: | , , , , , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
John Wiley & Sons, Inc.
2022
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Materias: | |
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 |