Dravet's syndrome research
We are a reference center in the research of this disease
Dravet syndrome is a rare genetic disease of childhood. It is a congenital brain disease that causes severe epilepsy, hyperactivity, intellectual impairment and movement disorders. Its treatment does not respond well to conventional drugs.
In most cases it is caused by mutations in a large gene (SCN1A) involved in the propagation of nerve stimuli in the brain.
At Cima we research Dravet syndrome focusing on the development and innovation of gene therapy treatments for a type of neurons related to the disease. We work in collaboration with the Clínica Universidad de Navarra to optimize these therapies and reach patients as soon as possible.
We dream of curing Dravet's syndrome
We seek to overcome resistance to current treatments and improve therapeutic options.
To deepen our understanding of the disease and develop more effective and safer inhibitory neuron-specific gene therapy vectors.
We seek to optimize the delivery and production of these specific vectors for potential clinical application.
We work closely with researchers and clinicians to rapidly translate findings from the laboratory to the patient.
REFERENCES IN DRAVET SYNDROME RESEARCH
Research Group in Gene Therapy of congenital diseases with neurological involvement.
Reference team in the research of encephalopathies specialized in the development of high-capacity adenovirus, a less toxic gene vehicle capable of harboring extensive DNA sequences and maintaining its function for long periods of time in the organism. Its objective is to design specific vectors for a specific type of neurons, safer and more effective, and to optimize their development for clinical application.
It is part of the Gene Therapy and Regulation of Gene Expression Program.