Protein Delivery

Assembling proteins for delivery

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Science  25 Mar 2016:
Vol. 351, Issue 6280, pp. 1412-1413
DOI: 10.1126/science.351.6280.1412-f

One of the challenges facing scientists who wish to one day use genome editing to treat disease is how to efficiently deliver proteins into cells. Wang et al. report one new strategy in which they combine positively charged synthetic lipids with negatively charged proteins to drive the self-assembly of nanoparticles that can efficiently enter cells. The hydrophobic tails of the lipids include a disulphide bond. The reductive environment inside the cell causes this bond to break, which enhances protein release from the nanoparticles. Delivering supercharged Cre recombinase enzyme or anionic Cas9:single-guide RNA resulted in efficient gene recombination and genome editing in cultured human cells and mouse brains, suggesting the promise of this technique.

Proc. Natl. Acad. Sci. U.S.A. 10.1073/pnas.1520244113 (2016).

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