Modeling Human Diseases with Stem Cells May Preempt Them

June 28, 2016 – Using stem cells to cure metabolic diseases in humans is the goal of Professor Qiurong Ding, Institute for Nutritional Science, Shanghai, China. Ding who spoke this week to an international economic forum in Tianjin, China, featuring young scientists, believes that studying Type 2 Diabetes, heart disease, inherited high cholesterol and obesity will lead to gene therapy treatments that will preempt these conditions by altering DNA.

Ding is using CRISPR/Cas9 to target genes associated with these diseases. She states “heart disease and high cholesterol are associated with certain gene mutations. We introduce these gene mutations into human pluripotent stem cells….” By doing this she and her team of researchers study the mutations and find ways to treat them in a petri dish. She has been doing it successfully with mouse genes which brings the question up, when will we be able to do it with humans?

Ding believes we are still a few years away from trying this on humans largely because of potential unintended consequences should a therapy impact more than the disease it targets. But the advantages of editing an existing genome are self-evident. It’s a permanent fix.

What Ding is doing is far from eugenics and designer babies although one could see the technology being used in this way. She restricts her research to non-reproductive tissues, liver and muscle cells. Altering the genome of a parent to correct a congenital disease (congenital means born with) does not protect future generations from inheriting the disease since sperm and egg cells are not within the realm of Ding’s research.

But that is coming as embryologists look at ways of intervening early in the development process to alter an inheritable genetic defect to ensure a healthy baby is born.

Ding believes we can eliminate almost all genetic diseases with this technology and line of research. And ultimately she believes all disease can be eliminated through gene editing.

You can read an interview given by Ding at the World Economic Forum at the link provided here.