Effective Small Interfering RNA Therapy to Treat CLCN7-dependent Autosomal Dominant Osteopetrosis Type 2

In about 70% of patients affected by autosomal dominant osteopetrosis type 2 (ADO2), osteoclast activity is reduced by heterozygous mutations of the CLCN7 gene, encoding the ClC-7 chloride/hydrogen antiporter. CLCN7G215R-, CLCN7R767W-, and CLCN7R286W-specific siRNAs silenced transfected mutant mRNA/...

Full description

Bibliographic Details

Published in:	Molecular Therapy: Nucleic Acids
Main Authors:	Mattia Capulli, Antonio Maurizi, Luca Ventura, Nadia Rucci, Anna Teti
Format:	Article
Language:	English
Published:	Elsevier 2015-01-01
Subjects:	bone disease bone resorption gene silencing osteoclast osteopetrosis siRNA therapy
Online Access:	http://www.sciencedirect.com/science/article/pii/S2162253116300348