Gene Therapy Using Recombinant AAV Type 8 Vector Encoding TNAP‐D10 Improves the Skeletal Phenotypes in Murine Models of Osteomalacia

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...

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Bibliographic Details
Main Authors: Flavia Amadeu de Oliveira, Fatma F. Mohamed, Yuka Kinoshita, Sonoko Narisawa, Colin Farquharson, Koichi Miyake, Brian L Foster, Jose Luis Millan
Format: Article
Language:English
Published: Oxford University Press 2023-01-01
Series:JBMR Plus
Subjects:
ANALYSIS/QUANTITATION OF BONE
ANIMAL MODELS
BONE QCT/MICRO‐CT
DENTAL BIOLOGY
DISEASES AND DISORDERS OF/RELATED TO BONE
GENETIC ANIMAL MODELS
Online Access:https://doi.org/10.1002/jbm4.10709

Internet

https://doi.org/10.1002/jbm4.10709

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