Article
Inhibition of PCSK9 with polypurine reverse hoogsteen hairpins: A novel gene therapy approach
11 May 2025
Abstract excerpt
PCSK9 is a therapeutic target for hypercholesterolemia. Though different strategies to inhibit PCSK9, such as monoclonal antibodies, small molecules, or nucleic acid drugs are available, the need for safer and inexpensive interventions remains. We developed a time-, cost-, and resource- efficient silencing system using Polypurine Reverse Hoogsteen (PPRH) hairpins to target PCSK9 . To achieve PCSK9 silencing, we...
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