A scientific team from the University of Chicago has solved several challenges that limited the possibilities of gene therapy and have shown that a new technique based on skin transplantation can make a wide range of these therapies viable to treat various human diseases . In an article published in Cell Stem Cell , they describe this new form of gene therapy administered through skin grafts to treat two very common diseases among the population: obesity and type 2 diabetes .
The group of researchers, led by Xiaoyang Wu of the University of Chicago, applied the editing technique known as CRISPR to create skin grafts from stem cells that produced a hormone capable of regulating blood sugar levels. The aforementioned technique ( Clustered Regularly Interspaced Short Palindromic Repeats : grouped and regularly interspaced short palindromic repeats) allows the genome of any living being to be easily modified . Then, they transplanted these grafts into diabetic mice and four months later they found that the insulin resistance of the animals had disappeared and even the weight gain caused by a high-fat diet had been limited . In a similar test carried out on human skin, the results were found to be similar. According to the authors of the study, this way of applying gene therapy is more efficient because the skin is the largest organ we have (it is throughout the body) and it is one of the simplest and cheapest tissues to produce from stem cells. at the laboratory.
The genetic modification applied in this study consisted of introducing a mutation in the GLP1 gene, which produces the hormone that regulates insulin , to increase its period of activity. In addition, the researchers added an antibody fragment to the gene to make it stay in the blood for longer. Finally, they applied a mechanism to get the GLP1 gene to produce more of the hormone when the mice took an antibiotic.
Wu and his colleagues see this method as a prototype test for introducing different gene therapies into the body. “It does n’t cure diabetes, but it does provide a potentially safe, long-term tool that can help people with diabetes and obesity keep their glucose levels more balanced, ” says Wu.
This team of scientists have focused on diabetes because it is a fairly common disease and not a skin disease that can be treated by applying a specific strategy with certain proteins.
But the case of diabetes will not be the only one in which the Chicago researchers intend to apply this technique. Wu believes it could also be used to transport therapeutic proteins or replace missing proteins in people with genetic defects , and it even has the potential to be used as a metabolic drain capable of removing various toxins.
In any case, the evolution of gene therapies is proof that the journey between basic laboratory research and real application in hospitals is long. More than two decades after the first laboratory studies, this way of treating the disease begins to become a real alternative, but some details, ethical objections and economic costs are still pending questions.
PHOTO CAPTION: Genetically modified mice gain less weight than others on a high-fat diet
Wu Laboratory, the University of Chicago