CRISPR Delivers Proteins, Not Genes | AIChE

CRISPR Delivers Proteins, Not Genes

March
2017

CRISPR is a promising gene-editing method that uses bacterial DNA to alter a cell’s genome. It has already been used to edit genes that were then injected into humans with aggressive lung cancer as part of a clinical trial in Chengdu, China. But although CRISPR holds promise for preventing and treating disease, researchers have raised safety concerns about the method. The most pressing dangers are that the system could edit the genome in the wrong place, and that the bacterial genetic machinery could cause an immune response.

A new nanoassembly method could overcome those concerns by delivering proteins, rather than the genes that prompt the cell to make those proteins, into the target cell. The method provides a gene-editing treatment that will vanish after it alters the cell, preventing rogue edits or immune responses later, says Vincent Rotello, a professor of chemistry at the Univ. of Massachusetts at Amherst.

CRISPR/Cas9 makes use of a bacterial defense system that splices portions of a foreign invader’s genome between genetic sequences...

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