First Patients Treated in U.S. CRISPR Study

The University of Pennsylvania has launched a trial of CRISPR-based gene editing in humans, and the first patients have been treated with the powerful – and controversial – technique, according to NPR. CRISPR is well established as a research tool, but this represents the first tangible step towards using CRISPR in the clinic to treat disease.

CRISPR allows scientists essentially to “cut out” and shut off a targeted gene and then re-introduce a version of the gene with altered or improved function. The University of Pennsylvania study involves removing patients’ immune system cells, genetically modifying them in the lab to target cancer cells, then re-infusing the modified cells. Two patients – one with multiple myeloma and one with sarcoma – have undergone infusion of CRISPR-edited immune cells. Both patients had disease that had relapsed following standard treatments. Eventually, the researchers hope to treat 18 patients in this study.

CRISPR and gene-editing has received increasing attention in the past year, following reports that a Chinese scientist had used CRISPR to edit a gene in the germline of human embryos to confer resistance to HIV infection. Recently, a panel of the World Health Organization requested a moratorium on all germline gene-editing experiments in humans, calling this type of research “irresponsible” without a central registry of research and other precautionary measures.

Several other human trials of CRISPR gene editing in a variety of conditions are planned and enrolling participants, including two studies of gene editing to correct the genetic defects that cause sickle cell disease and beta thalassemia.

Source: NPR, April 16, 2019.