• Nature
    Comprehensive Ophthalmology

    Scientists from Sichuan University in China are the first to test CRISPR-associated protein-9 nuclease (CRISPR–Cas9) modified cells in humans, as part of a clinical trial that will focus primarily on safety.

    Scientist in the United States are also planning a trial using CRISPR to target 3 specific genes to treat various cancers. Dr. Carl June, an immunotherapist from the University of Pennsylvania in Philadelphia serving as scientific adviser to the trial, said he anticipates an international race to get gene-edited cells into the clinic. His trial is set to begin in early 2017.

    "I think this is going to trigger ‘Sputnik 2.0’, a biomedical duel on progress between China and the United States, which is important since competition usually improves the end product,” Dr. June said.

    The gene-editing technology is based on the bacterial system of clustered regularly interspersed short palindromic repeats (CRISPR) that can effectively repair or knockout without reproduction for each new target, making it simpler and more cost effective than previous gene editing methods. Its specificity depends largely on a guide RNA that can be programmed to target different genomic loci, allowing for fast, accurate DNA editing.

    CRISPR-Cas9 has the potential to cure a host of diseases, including corneal diseases and retinitis pigmentosa, but oncology appears to be the favored early target. The Chinese team led by oncologist Lu You plan to treat 10 patients with metastatic non-small-cell lung cancer, according to the report in Nature.

    Lu and his team removed immune cells from the recipient’s blood and disabled a gene using CRISPR-Cas9. After culturing the edited cells to increase their number, they injected them back into the patient, with the hope that the edited cells will attack and defeat the cancer.

    Lu reports that the treatment went smoothly and a second injection is planned, but declined to give further details due to patient confidentiality. Subjects will receive 2, 3 or 4 injections and will be monitored for 6 months for serious adverse effects, such as an unwanted autoimmune response.