U.S. CRISPR Study in Humans Gets Underway

The first patients enrolled in a trial of CRISPR-based gene editing at the University of Pennsylvania 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 toward using CRISPR in the clinic to treat disease.

The 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.

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-based 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.