Kormann Lab is dedicated to provide efficient and safe therapies for severe genetic diseases of the lungs, such as Cystic Fibrosis, and of blood stem and immune cells.
Cystic Fibrosis (CF)
is driven by mutations in the underlying disease-conferring gene, CF transmembrane conductance regulator (CFTR), and is fatal with no available cure . CF is the most prevalent life-limiting autosomal recessive disease in Caucasian populations, occurring in 1/2,500 newborns and affecting more than 70,000 people worldwide .
Disease-causing CFTR mutations result in impaired chloride secretion and hyper-absorption of sodium across the epithelia, leading to severe chronic lung disease. Most patients do not survive beyond a mean age of 30-40 years. Numerous attempts have been made to develop gene therapeutic approaches for the treatment of CF, using viral or non-viral vectors to supplement cells of the lung with a functional copy of CFTR.
However, these strategies remain unsuccessful, largely due to limitations in efficiently targeting the lung. Now, researchers - due to the complexity of DNA repair - try to fix the faulty CFTR messenger RNA (mRNA). However, even if successful in the clinic, this would still mean daily or hourly inhalations, due to the rapid decay of mRNA.
To circumvent that, we are focussing on a possibility to fix CFTR at its genomic locus , in lung and intestines, therefore providing real cure of the disease. Done in stem cells this could be the breakthrough that so many patients desperately need.
Jun.-Prof. Dr. rer. nat. Michael Kormann
Principal Investigator, Assistant Professor for Translational Genomics and Gene Therapy in Pediatrics
After receiving his diploma in biology of the Technical University of Munich, Dr. Kormann did his PhD in asthma genetics, receiving excellent doctoral training in the group of Michael Kabesch at the Ludwig-Maximillians-University of Munich, which he finished with summa cum laude. He then had his postdoctoral training in the laboratory of BioFuture Award winner Carsten Rudolph. In the months since, he then changed to the University of Tübingen as a junior group leader. Two Nature Biotechnology papers (2011 & 2015) describe the first therapeutic use of modified messenger RNA (transcript therapy) and therapeutic genome editing in mouse lungs. In 2011 he became one of the youngest Junior Professors at the University of Tübingen. He was awarded with the prestigious Maurizio Vignola Award for Innovation in Pneumology and the Klosterfrau Grant for Research of Airway Diseases. In 2014 he received an ERC Starting Grant, one of the most renowed european grants, for research on Cystic Fibrosis and Surfactant Protein B deficiency in vivo gene correction.
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