Gene Therapy via Lipid Nanoparticle 2

Gene Therapy via Lipid Nanoparticle 2

Collections: Nano-based Drugs

Corina MacIsaac, Jacob Witten, Allen Jiang, Idris Raji, Robert Langer, Daniel Anderson

Koch Institute at MIT

We are developing gene therapy for the lung, which could treat diseases ranging from lung cancer to asthma. The major limiting factor for lung gene therapy has been the ability to deliver a gene into cells without causing toxicity. Here, we used lipid nanoparticles (LNPs) invented in our lab to deliver a gene that causes cells to turn red, to test how effective our LNPs were. We found very good delivery to a wide variety of lung cells, including important lung cell types such as club cells (green) and ciliated cells (purple border).

a stripe of red rings the edge of a mass of blue cells, shaped collectively like the open jaws of a cartoon monster

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