Gene Therapy in Neurological Diseases

"Our goal is to implement advanced treatments, such as gene therapy, to slow the progression of the most prevalent neurodegenerative diseases, such as Alzheimer's and Parkinson's disease."

DR. ANA Mª GARCÍA OSTA
RESEARCHER. GENE THERAPY IN NEUROLOGICAL DISEASES RESEARCH GROUP

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Gene Therapy in Neurological Diseases

Our Gene Therapy in Neurological Diseases Research Group studies the most prevalent and disabling neurodegenerative diseases, such as Alzheimer's disease (AD) and Parkinson's disease (PD).

We aim to identify the genes responsible for synaptic dysfunction and neuronal death to develop treatments primarily based on gene therapy. These therapies will be focused on modifying genes in the brain to correct the identified dysfunctions to slow or stop the progression of the disease.

To carry out our research, our laboratory uses transgenic animal models that express the proteins that constitute the histopathological markers of each disease (beta-amyloid or synuclein) or adeno-associated viruses to generate neuronal accumulation of cytotoxic proteins such as tau or neuromelanin.

Dr Ana García Osta

GROUP LEADER

	+34 948 194 700 \| Ext. 812023
	agosta@unav.es

Gene Therapy in Neurological Diseases
Research Group Objectives

To study the mechanisms and molecular basis of cognitive and motor impairment in neurodegenerative.

To identify new targets and therapeutic genes.

Designing advanced therapies, especially gene therapy, aimed at slowing the progression of neurodegenerative diseases.

Advanced therapies for neurodegenerative diseases

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Alzheimer's is a devastating disease for which there is no cure. New strategies are needed to identify effective disease-modifying therapies for Alzheimer's disease. In our laboratory, we have analyzed the brains of resilient individuals, who retain their ability to learn despite severe pathology, to identify new therapeutic targets.

We have identified a new potential therapeutic target for Alzheimer's disease: the PLA2G4E protein, which could make us "resilient" to Alzheimer's disease. Administration of this enzyme through gene therapy to animal models of Alzheimer's disease protects them from dementia.

We also work in collaboration with the Department of Pharmaceutical Chemistry and Technology and the Immunology program for the development of advanced therapies with neuroimmunomodulatory effect to slow down the development of neurodegenerative diseases such as Alzheimer's and Parkinson's.

Lines of research

To study the mechanisms and molecular basis of cognitive and motor impairment in neurodegenerative diseases such as Alzheimer's disease and Parkinson's disease.

To identify new targets and therapeutic genes with which to design advanced therapies, especially gene therapy, aimed at slowing the progression of neurodegenerative diseases.