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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Main Authors: | Mattia Capulli (Author), Antonio Maurizi (Author), Luca Ventura (Author), Nadia Rucci (Author), Anna Teti (Author) |
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Format: | Book |
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Elsevier,
2015-01-01T00:00:00Z.
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