Gene Therapy Using Recombinant AAV Type 8 Vector Encoding TNAP‐D10 Improves the Skeletal Phenotypes in Murine Models of Osteomalacia
ABSTRACT 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. PHOSP...
Main Authors: | Flavia Amadeu de Oliveira, Fatma F. Mohamed, Yuka Kinoshita, Sonoko Narisawa, Colin Farquharson, Koichi Miyake, Brian L Foster, Jose Luis Millan |
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Format: | Article |
Language: | English |
Published: |
Oxford University Press
2023-01-01
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Series: | JBMR Plus |
Subjects: | |
Online Access: | https://doi.org/10.1002/jbm4.10709 |
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