RNA interference therapy for autosomal dominant osteopetrosis type 2. Towards the preclinical development.
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DOI: 10.1016/j.bone.2018.02.031
Abstract: Autosomal Dominant Osteopetrosis type 2 (ADO2) is a rare bone disease characterized by dense and brittle bones due to impairment of osteoclast bone resorption. Dominant negative mutations of the CLCN7 gene affect about 70% of… read more here.
Keywords: osteopetrosis type; dominant osteopetrosis; rna interference; bone ... See more keywords