Gene editing could be used to control diseases like heart disease in people, stem cell researcher Qiurong Ding from the Institute for Nutritional Sciences in Shanghai tells Scientific American.
Ding currently uses stem cells to model certain human diseases like type 2 diabetes, heart disease, obesity, and inherited high cholesterol. Her lab introduces mutations linked to heart disease and high cholesterol into pluripotent stem cells to study them and possible treatments. "We needed a model that could mimic what was happening in a human patient," she tells Sciam, adding that "[b]ecause stem cells proliferate and can turn into many different types of somatic cells, they allow us to study many different diseases and test different drugs."
At the same time, Ding also uses gene therapy to treat high cholesterol in mice. This work, she says, is builds off the discovery of a group of people in Texas who, despite eating a high-fat, high-sugar diet, had very low cholesterol due to a gene mutation. Using CRISPR, she's introduced this mutation into humanized mice. This mutation appears to have led to decreased cholesterol in the mouse model.
She adds that many diseases may be able to be prevented in the future through editing of somatic cells. " Of course some diseases are caused by lifestyle, but those can be prevented," Ding says. "As for genetic diseases, that's the goal we ultimately want to achieve — to eliminate them."