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The Onpattro story and the clinical translation of nanomedicines containing nucleic acid-based drugs

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The regulatory approval of Onpattro, a lipid nanoparticle-based short interfering RNA drug for the treatment of polyneuropathies induced by hereditary transthyretin amyloidosis, paves the way for clinical development of many… Click to show full abstract

The regulatory approval of Onpattro, a lipid nanoparticle-based short interfering RNA drug for the treatment of polyneuropathies induced by hereditary transthyretin amyloidosis, paves the way for clinical development of many nucleic acid-based therapies enabled by nanoparticle delivery.

Keywords: acid based; onpattro story; clinical translation; story clinical; nucleic acid; translation nanomedicines

Journal Title: Nature Nanotechnology
Year Published: 2019

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