Successful Gene Therapy in Utero for Lethal Murine Hypophosphatasia

Hypophosphatasia (HPP), caused by mutations in the gene ALPL encoding tissue-nonspecific alkaline phosphatase (TNALP), is an inherited systemic skeletal disease characterized by mineralization defects of bones and teeth. The clinical severity of HPP varies widely, from a lethal perinatal form to mil...

Full description

Saved in:
Bibliographic Details
Main Authors: Sugano, Hanako, Matsumoto, Tae, Miyake, Koichi, Watanabe, Atsushi, Iijima, Osamu, Migita, Makoto, Narisawa, Sonoko, Millán, José Luis, Fukunaga, Yoshitaka, Shimada, Takashi
Format: Artigo
Language:Inglês
Published: Mary Ann Liebert, Inc. 2011
Subjects:
Research Articles
Online Access:https://ncbi.nlm.nih.gov/pmc/articles/PMC3327603/
https://ncbi.nlm.nih.gov/pubmed/22133046
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/hum.2011.148
Tags: Add Tag
No Tags, Be the first to tag this record!

Similar Items