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.

Dra. Ana Mª García Osta


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   Research profile

Gene Therapy in Neurological Disorders Research Groups

 José Luis Lanciego José Luis Lanciego
Principal Investigator
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Ana García Osta Ana García Osta
Principal Investigator
Montse Arrasate Montse Arrasate
Principal Investigator

Main objective of the program

Icono naranja de un cerebro

The ultimate goal is to identify and validate new therapeutic targets and genes that can be modified through  gene therapy, capable of halting the progression of these diseases by preventing neuronal death, restoring synaptic transmission, and normalizing the functioning of neural circuits.


Cutting-edge translational research

New Therapies and Company Relationships

To drive translational research and facilitate the development of new therapies that address the most urgent clinical needs in the field of Neurosciences, we collaborate closely with other researchers in the DNA and RNA Medicine division and the Immunology program at CIMA, as well as with the Department of Pharmaceutical Technology and Chemistry at the University of Navarra. In addition, we maintain a close collaboration with neurologists from CUN and HUN.


Our research has resulted in several patents. Likewise, the experience of our research team with animal models, ranging from rodents to primates, has facilitated the collaboration with national and international companies to carry out the preclinical development of therapeutic viral vectors. Our goal is to move towards clinical trials in patients affected by neurodegenerative diseases.

<p>Fachada del Centro de Investigaci&oacute;n M&eacute;dica Aplicada (CIMA)</p>

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Thanks to the generosity of many people, the Cima Universidad de Navarra is a reality that strives to offer therapeutic solutions to achieve personalized medicine for patients.