BREAKTHROUGH IN GENE THERAPY RESEARCH FOR HEREDITARY MUSCLE DISEASES
Research into promising treatment disease of Duchenne published in prestigious journal Nature Communications
Wednesday, January 30, 2019 — Professors Marinee Chuah and Thierry VandenDriessche of the Vrije Universiteit Brussel (Belgium) have developed a new and more efficient gene therapy for Duchenne's disease. The new study has now been published in Nature Communications. Through gene therapy, genes are introduced in muscle cells in order to cure the underlying disease. The disease affects 1 in 3500 boys and is one of the most common hereditary diseases. This new gene therapy also offers new perspectives for the treatment of other muscle disorders.
Prof. Marinee Chuah and prof. VandenDriessche (Free University of Brussels, Belgium) currently lead the only research group in Belgium that has focused on developing gene therapy for over 20 years. They are also affiliated with the KU Leuven. DNA is the genetic material that consists of a sequence of 3 billion chemical letters. If an error occurs in one of these letters, a hereditary transmissible disease occurs. This genetic typing error can be life-threatening and hangs like a sword of Damocles over the head of the patients who are born with this disorder. Professor VandenDriessche: "Our research is inspired by a philosophical-humanistic vision, in which one cannot accept that human suffering is predestined by what is written in the DNA". In patients suffering from Duchenne's disease, the condition is due to a defect in the dystrophin gene on the X chromosome. As a result, the muscles inevitably deteriorates and the patients are no longer able to move independently. In addition, patients with Duchenne suffer from heart problems and have breathing difficulties They eventually succumb from heart and lung failure at a relatively young age. In the case of inherited transmissible diseases, gene therapy can be used to try to correct the cause of the disease at the DNA level. Nevertheless, it is quite a challenge to develop effective and safe gene therapy for such hereditary diseases. The teams of Prof. Marinee Chuah and Prof. VandenDriessche have now realized a breakthrough in research into gene therapy for hereditary muscle diseases, and in particular Duchenne's disease. They have developed a kind of 'molecular turbo engine' that significantly improved the effectiveness and safety of gene therapy for Duchenne's disease. The promising strategy was tested in experimental animals with Duchenne's disease. Prof. dr. Chuah: "Our new gene therapy technology prevented the decay of the muscles that cured the disease in the Duchenne mice. This is the first step toward future clinical applications in patients suffering from Duchenne”. Their promising research was published in the leading scientific journal Nature Communications.
Broad implications
Moreover, their research has important implications for other hereditary muscle diseases such as Steinert's disease. Not only is it possible to prevent muscle decay via this gene therapy, but it can be used to apply genetic corrections to the DNA in patient’s cells by means of gene editing with CRISPR/Cas technology with unprecedented nanomolecular precision. The research was funded in part by the Fund for Scientific Research and the King Baudouin Foundation and the European Union.
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Prof. Marinee Chuah
Prof. Thierry VandenDriessche
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0477/52 96 53