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Most approved gene therapies today, including those involving CRISPR-Cas9, work their magic on cells removed from the body, after which the edited cells are returned to the patient.
<p>By attaching two kinds of antibody fragments to membrane-derived globules, UC Berkeley researchers selectively delivered CRISPR-Cas9, guide RNA and a transgene to T-cells in living mice to create CAR T-cells. While this technique could make it easier to create CAR T-cells <em>in vivo</em> for cancer therapy, the ultimate goal is to selectively target CRISPR-filled "enveloped delivery vehicles" to any type of cell and avoid the <em>ex vivo</em> cell editing now used in current gene therapies.</p>