Oreos are two-year-old Nate Liao’s current favorite snack. That may seem insignificant – but it’s actually remarkable he can eat solid food. Nate and his 5-year old brother Jake have a rare genetic condition called Epidermolysis Bullosa – or E-B.

If you can picture Velcro – one side would be your muscle. One side is your skin. There’s actually hooks that are actually anchoring into the muscle tissue holding the skin on. In the form my boys have, we don’t have those. So basically, the skin just shifts around or it peels right off.

E-B makes everyday life a challenge. Nate and Jake must be wrapped with bandages to protect the skin and allow wounds to heal. E-B makes skin so delicate, a seam on a piece of clothing can cause the skin to blister and come off. The risk of life-threatening infections or cancers is so great, few E-B patients reach the age of 30. Eating and drinking is difficult. Anything that goes in their mouth causes tissue damage.

But there is hope on the horizon for the Liao family and the thousands of families who share their struggle with E-B. A team of researchers at the University of Minnesota has discovered a cure.

It is very unusual to say you have a cure after just one patient. However, what’s extraordinarily different in this case is that we’ve been able to demonstrate by electron microscopy, also by protein analysis, and also by looking at the patient that we made a fundamental change. What was not present at all to begin with, is now present.

What’s now present is collagen VII, the key to the form of E-B that affects Jake and Nate. It’s a protein that allows skin to hook into muscle. Researchers believed if they could correct the collagen VII defect, they would reverse the effects of E-B.

Here’s how they did it. Through trial and error, Dr. Jakub Tolar treated mice genetically altered to have E-B with different types of healthy bone marrow and cord blood cells. Some of the mice lived and their blisters cleared up because they were producing collagen VII, the protein missing in the Liao boys.

So once we had the results in an animal model, we had the proof of principle, that a cell population from the marrow could correct E-B. We took that strategy first proved in a mouse and applied that to a clinical trial. In October of last year, Nate received a transplant of cells from bone marrow and cord blood from his brother Julian, who is a coincidental perfect match and is not affected by E-B. Seven months later, Nate’s collagen VII levels are near normal and his skin is noticeably less blistered.

Before when you looked at Nate, he looked like his skin was kind of thin. In parts, he almost kind of looked translucent. It appears that the layer that was missing, is there.

We truly believe, that having now corrected the collagen type VII deficiency that he will no longer have that risk of cancer, that he will no longer have the risk of mutilating scarring, that his quality of life will be enhanced tremendously.

To be able to throw them in a bathtub, put them in pajamas and put them to bed, would be an amazing thing. Without having to worry about all of the nutritional needs and the tube feeds, and the bandages… that would be an amazing thing.

The hope offered by this cure doesn’t end with the Liao’s. The knowledge gained will allow researchers at the University of Minnesota and elsewhere to explore more disease areas and develop more cures.