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Pharmacologist Christian Grimm has created a new study on the causes of childhood dementia and has now received the NCL Research Award. It is about hereditary neuronal ceroid lipofuscinosis, which is currently incurable.
A deadly disease
In this form, the cell organelles do not work as they should. Damage in various proteins of these lysonsomes triggers several variants of the disease, but until now the researchers knew little about how these proteins work.
Blindness, epilepsy and decay
The nerve cells of those affected die, they lose their eyesight to the point of blindness, suffer from impaired consciousness, the musculoskeletal system no longer works, they twitch uncontrollably and so far they die inevitably because the functions that maintain the organism fail. Increased mortality from epilepsy alone is just one reason why these children die early.
The CLN3 protein
Grimm is now trying to decrypt the CLN3 protein. This protein causes the disease that begins in primary school and kills the nerve cells - in the end the patient dies. CLN3 occurs in the lysosomes that break down biomolecules. Grimm suspects that CLN3 is an essential channel for ions and calcium. He is now investigating these functions using the so-called patch clamp method.
The patch clamp technique
Patch-Clamp makes it possible to observe the electrical behavior of CLN3 proteins. To do this, the scientists suck in a part of the membrane with a micropipette, put on a microelectrode and send current into the ion channels. This is how they show whether the channel is being used or not.
In this way, Grimm wants to demonstrate whether CLN3 is an ion channel and also recognize the molecules that activate CLN3. To do this, the researchers also need systematic screening.
New agents for therapy
CLN3 is often hardly available in those affected. Grimm would therefore like to find substances that can replace the protein. The scientists have already discovered some substances that are suitable for this. (Dr. Utz Anhalt)