Giving Compass' Take:

· To help treat inherited genetic diseases, Pedro Belda Ferre, Postdoctoral Scholar in Pediatrics at the University of California San Diego, explains that engineering bacteria to produce the needed substance can be a great way to help fight these illnesses.

· Is this a safe way to treat diseases? What are some other ways to treat inherited genetic diseases?

· Interested in learning more on the potential of bacteria? Check out his article about how bacteria is helping fight pollution. 

A pill containing millions of bacteria ready to colonize your gut might be a nightmare to many. But it may become an effective new tool for fighting disease.

In many inherited genetic diseases a mutated gene means that an individual cannot make a vital substance necessary for their body to grow, develop or function. Sometimes this can be fixed with a synthetic substitute — a pill — that they can take daily to replace what their body should have made naturally. People with a rare genetic disease called phenylketonuria (PKU), lack an enzyme that is essential for breaking down protein. Without it, toxic chemicals build up in the blood and can cause permanent brain damage.

Fortunately, the fix is easy. Physicians treat the disease by putting their patients on a super low-protein diet for the rest of their life. Indeed, because the fix was so simple PKU was the first disorder for which newborn babies were routinely screened, beginning in 1961, by analyzing a drop of blood collected from a prick on the baby’s heel.

But imagine how challenging it can be to measure everything you eat during your entire life. To cure PKU researchers are currently exploring new treatment strategies. One involves using gene-editing tools to correct genetic mutations. However, the current technology is still risky; there is a chance of disrupting other genes and causing collateral damage to patients.

What if one could replace the broken gene without affecting the patient’s genome? That’s exactly what researchers at the Cambridge, Massachusetts-based biotech company Synlogichave done. They decided that rather than meddling directly with the human genome, they would introduce the therapeutic genes directly into the naturally occurring bacteria that reside in the human gut. These genetically modified bacteria would then produce the enzymes that PKU patients lacked and break down the proteins into non-toxic products.

Read the full article about engineering bacteria to treat genetic diseases by Pedro Belda Ferre at The Conversation.