Cystic Fibrosis: research on a new therapeutic target in Germany

Bypassing the defective channel by activating an alternative channel

Cystic fibrosis is one of the most common congenital metabolic diseases in the European population. With the help of the Air Liquide Foundation, the German cystic fibrosis association, Mukoviszidose e.V, provides support to Dr. Ulrich Martin from the Hannover Medical School on a new therapeutic approach to cystic fibrosis.

A genetic disease

Cystic fibrosis is caused by a genetic mutation that changes the protein responsible for regulating the transport of chloride through cell membranes. Chloride controls the hydration of the mucus. This malfunction leads to more viscous mucus that accumulates in the respiratory and digestive tracts, providing an ideal breeding ground for bacteria and germs that cause inflammation.

Cystic Fibrosis mainly affects the lung and bronchial system and the most common consequences are recurrent pneumonia, chronic cough, severe digestive disorders and liver damage.

Mukoviszidose e.V

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_{Research team: Ruth Olmer, Sylvia Merkert & Ulrich Martin © Karin Kaiser - Hannover Medical School}

Issue of mutation-specific therapies

There are more than 2,000 genetic mutations which can cause a change in the protein responsible for the transport of chloride. Available therapies for cystic fibrosis are mutation-specific and not all patients can benefit from them.

An alternative channel

In the Hannover Medical School, Dr. Ulrich Martin is studying on an alternative chloride channel which could restore the malfunction caused by the change in the protein. In that way, the defective channel would be bypassed.

Therapies based on this alternative channel could treat all patients of cystic fibrosis as they would not be mutation-specific.

The purpose of Dr. Ulrich Martin’s research is to exactly identify the role of TMEM16A. More specifically, it should be determined whether an activation or inhibition of TMEM16A is beneficiary.

The contribution of the Air Liquide Foundation

The Air Liquide Foundation allocation of EUR 50,000 is used for the purchase of material resources for the research project.