Compositions for PCSK9 gene editing and methods of using same for treatment of disease

Abstract

Disclosed herein are methods of disease treatments by administering to a subject in need thereof a pharmaceutical composition comprising gene editing compositions. The methods disclosed herein can provide durable in vivo editing of PCSK9. More particularly, the compositions disclosed herein are capable of in vivo editing PCSK9 gene in humans and reducing PCSK9 protein and LDL-C for a potentially transformative treatment of cardiovascular disease, the leading cause of death in the United States and world-wide. Various aspects of the compositions, use and preparation are disclosed, including clinically supported therapeutically effective and safe human dosing of the compositions.

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