While gene editing is remarkably precise in finding and altering genes, there is still no way to target treatment to specific locations in the body. The treatments tested so far involve removing blood stem cells or immune system T cells from the body to modify them, and then infusing them back into a patient. Now, researchers at Tufts University have, for the first time, devised a way to directly deliver gene-editing packages efficiently across the blood brain barrier and into specific regions of the brain, into immune system cells, or to specific tissues and organs in mouse models. The researchers used lipid nanoparticles – tiny "bubbles" of lipid molecules that can envelop the editing enzymes and carry them to specific cells, tissues, or organs.
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