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Treating and Curing Disease Through Human Genome Editing

April 26, 2018
DNA defines who we are. And like anything else, sometimes there are mistakes – mutations in genes that can cause life-lasting conditions. But what if broken genes could be repaired? How many patients could we help in the years ahead? This is the promise and possibility of human genome editing.

While DNA modification techniques have existed for decades, recent developments in genome editing technologies have provided scientists and researchers with far more precise and efficient tools than ever before. As a result, 2017 marked the first genome editing clinical trials in humans initiated in the United States which are targeting a metabolic disorder known as Hunter Syndrome. And research is currently underway on clinical applications of genome editing technologies to treat genetic disorders like sickle cell disease, cystic fibrosis, congenital blindness, hemophilia, amyloidosis, and lysosomal storage disorders.

As we look beyond human health, these products can be applied to animal and plant DNA, as well as many organisms that are used in basic biological research. Applications in agriculture and animal health have the potential to deliver major advances to help feed the world.

To learn more about human genome editing, head to our Toolkit for Advocates and check out our infographic and issue brief on this important topic.