NIH Reporter #1R44HL184837-01A1

Non-viral gene therapy to treat and prevent atherosclerosis

Award for developing a non-viral gene therapy using lipid nanoparticles to treat and prevent atherosclerotic cardiovascular disease, aiming for a single-treatment commercial product.

Description

Development of a non-viral gene therapy vector that mitigates the development of atherosclerotic cardiovascular disease after a one-time administration, using lipid nanoparticles to deliver DNA efficiently to the nuclei of long-lived non-dividing cells, with proof-of-concept in mice and stable expression in non-human primates.

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