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

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/...

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

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http://www.sciencedirect.com/science/article/pii/S2162253116300348

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