Hepatic Diseases Gene Therapy

"Despite the progress made in the development of gene therapies for inherited liver diseases, the clinical translation of these strategies requires many improvements to increase their efficacy."

DR. GLORIA GONZÁLEZ

The Gene Therapy of Liver Diseases Group at Cima focuses its research on two lines of research:

Gene therapy of rare liver diseases of genetic origin: study of diseases whose origin is in the liver and in particular those hereditary diseases that can be cured by liver transplantation. We work to understand the pathophysiology of some of these rare diseases and to develop new therapies through strategies based on gene transfer and gene editing techniques that can offer solutions to these pathologies.

Study and development of new treatments for chronic viral hepatitis: we seek to deepen the knowledge of the pathology associated with infection by hepatitis B virus (HBV) and hepatitis Delta virus (HDV) to develop new treatments for these chronic infections.
To carry out this research, our group strives to improve the gene transfer vehicles, thanks to a better knowledge of the vector at the molecular level and of different aspects that affect its efficacy. On the other hand, thanks to the use of adeno-associated viruses as vehicles to transfer genes to the liver, we have developed animal mouse models that reproduce some of the most important characteristics of these diseases in humans.

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Objectives of the Research Group in
Gene Therapy in Hepatic Diseases

Acquire a deeper understanding of genetic liver diseases to improve the efficacy of gene therapy treatments.

Development of gene editing strategies for primary hyperoxaluria type I.

To develop new animal models of viral hepatitis infection that improve knowledge of molecular biology.

To develop new antiviral treatments for liver diseases.

Priority diseases in our research group

Hyperoxaluria

Primary hyperoxaluria type I (PH1) is a rare metabolic disease due to mutations in the AGXT gene, which encodes AGT. Patients have severe renal damage and the only curative treatment is transplantation.

FROM THE LABORATORY TO THE PATIENT

Cutting-edge translational research

The research team of the Gene Therapy Program is fundamentally basic, but with a very translational vision and maintains close collaborations with teams from the Clínica Universidad de Navarra.


Clinical
translation

Improvement of therapies developed for genetic diseases and cancer to provide solutions for patients.


Transfer DNA or RNA in vivo

Implementation of new gene therapy technologies using gene editing or non-viral vectors.


Long non-coding RNAs (lncRNAs)

Development of new expression regulation systems, as well as the identification of new therapeutic targets.

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

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Meet the research team

Scientific activity of the Biomarkers and New Therapeutic Targets in Lung Cancer Group