Gen eraser Are chronic diseases such as heart and kidney weakness soon to be curable?

Heal chronic diseases with new treatment approach

Chronic diseases often accompany sufferers throughout their lives and limit their quality of life and, not infrequently, their life expectancy. Current treatments are largely aimed at mitigating and slowing down the effects of the disease. One main reason why many chronic diseases can not be cured is that they have already brought about changes in the genome. They keep the disease alive. A German research team has now succeeded in simply erasing these epigenetic changes. They call their discovery a gene eraser.

Göttingen researchers have developed a new method for eradicating changes in the genetic material caused by chronic diseases. These changes are responsible for the disease process in many chronic conditions, such as heart failure or kidney failure. The study results were recently published in the renowned journal "Nature Communications".

The cause lies in the genes

Respiratory problems, even shortness of breath, chronic fatigue, water retention in the limbs - these are among other typical symptoms with which sufferers of heart or kidney weakness constantly struggling. With age, the complaints become more and more obvious as the performance of the organs decreases. The basis for these diseases are so-called epigenetic changes. These are modifications that subsequently change the genetic material (DNA) and have made some useless. Responsible for this are chemical processes, with which, for example, certain molecules were coupled to the DNA (DNA methylation).

Etching instead of cutting

The team around Professor dr. Michael Zeisberg of the University Medical Center Göttingen succeeded for the first time in reversing these pathological changes. The researchers further developed the technology known as "gene-scissors". "Rather than cut the DNA, a gene segment may by the modified technology now be cleaned as with an eraser on the modification," Zeisberg explained in a press release on the study results.

Just erase chronic diseases

As the researchers report, this new method can be used to correct DNA methylation and fibroblasts responsible for disease progression. Fibroblasts are fibrous or connective tissue cells that cause the regeneration and remodeling of connective tissues. If there is an epigenetic change, the fibroblasts also release epigenetically altered DNA, which can even be detected in the blood. "Potentially can thus be identified disease-causing epigenetic modification in the kidney or the heart with a simple blood test and then corrected with a tailored therapy," write the genes Xperts.

Personalized treatment of cardiac or renal insufficiency

This method constitutes a new approach to personalized treatment of heart failure and chronic renal failure. "The results of this study do hope that we have a further effective method for the treatment of heart failure is available in the future," adds Professor Dr. Gerd Hasenfuß, the chairman of the heart center. With this method, a wide range of applications is possible to regulate the activity of individual genes. The big advantage: You do not have to change the actual genetic sequence, but only undo harmful changes.

The DNA reset

The principle of gene scissors has been discovered in bacteria that release themselves from harmful viruses by directing a particular protein (Cas9) to any location in the DNA and severing the duplex at that site. "For our purposes, we have altered the Cas9 enzyme in a way that allows it to bind specific gene segments but no longer has the ability to cut them," explains Zeisberg. The researchers coupled the CAS9 protein with another enzyme called Tet3. The effect: Instead of cutting the DNA, pathogenic modification was removed and the original state of the DNA sequence restored.

So far only tested in the animal model

"The study demonstrated for the first time the successful application of this method in the animal model," said the research team. The researchers are currently working to make the modified enzyme more compact. After that, it should also be tested in human patients. (Vb)