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Bone-Targeting AAV-Mediated Gene Silencing in Osteoclasts for Osteoporosis Therapy

Yeon-Suk Yang, Jun Xie, Sachin Chaugule, Dan Wang, Jung‐Min Kim, JiHea Kim, Phillip W.L. Tai, Seok Kyo Seo, Ellen M. Gravallese, Guangping Gao, Jae‐Hyuck Shim

2020Molecular Therapy — Methods & Clinical Development49 citationsDOIOpen Access PDF

Abstract

is a promising gene therapy that can preserve bone formation and mitigate osteoporosis, while limiting adverse off-target effects.

Topics & Concepts

OsteoclastCathepsin KGene silencingOsteolysisActivator (genetics)OsteoporosisCancer researchBone resorptionGenetic enhancementMedicineRANK LigandRANKLCell biologyReceptorInternal medicineChemistryBiologyGeneDentistryBiochemistryBone Metabolism and Diseasesinterferon and immune responsesRNA Research and Splicing
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