Gene Therapy in Neurological Disorders

In recent decades, in parallel with the increase in life expectancy, neurodegenerative brain diseases, which particularly affect the elderly population, have become an increasingly heavy burden on society. Among these diseases, Alzheimer's and Parkinson's stand out as the most prevalent, primarily affecting cognitive and motor functions respectively, causing a devastating impact on their lives and those of their loved ones. Current treatments only offer symptomatic relief and do not halt the progression of the disease, underscoring the urgency of finding new therapies capable of slowing its development. Modifying genes associated with these diseases through gene therapy represents a radical change in their treatment.

The objective of the program of Gene Therapy for Neurological Diseases is to understand the mechanisms and molecular basis of cognitive and motor impairment in neurodegenerative diseases, specifically Alzheimer's disease and Parkinson's disease.  Based on this knowledge, we develop new therapeutic strategies that we test in various preclinical models of different pathologies. To achieve this, cellular models, transgenic animal models, and animal models generated using adeno-associated viruses are employed to recapitulate the pathological alterations as well as the cognitive or motor impairment observed in patients affected by these diseases.