Clinical pharmacology of siRNA therapeutics: current status and future prospects.

Expert review of clinical pharmacology(2022)

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摘要
Few siRNA-based products have been approved recently by the Food and Drug Administration (FDA) and other regulatory agencies after approximately 20 years following its discovery due to the associated limitations. The absorption, distribution, metabolism, and excretion of siRNA therapeutics are highly restricted by several obstacles, resulting in rapid clearance of siRNA-based therapeutic products from systemic circulation before reaching the cytosol of targeted cells. The siRNA therapeutics however are very promising in many diseases, including gene therapy and SARS-COV-2 viral infection. The design of suitable delivery vehicles and developing strategies toward better pharmacokinetic parameters may solve the challenges of siRNA therapies.
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关键词
Small interfering RNA (siRNA),bioavailability,bioconjugation,givosiran,lumasiran,patisiran,pharmacodynamic
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