Gene Therapy and Regulation of Gene Expression

Technological advances in the field of molecular biology in recent years have given us a more comprehensive understanding of the genetic material of living beings. This contains information that determines what we are. These advances have resulted in important achievements such as the description of the composition of the human genome.

Analysis of the human genome has been and remains key to our understanding of the molecular basis of very different pathologies, particularly in hereditary diseases. This understanding, together with the great strides achieved in the field of genetic engineering and the development of more efficient and safe gene transfer vectors, has resulted in great advances in the field of gene therapy (therapeutic strategies based on the transfer of genes), which has become a medical alternative in diseases that are currently incurable. Not just for diseases associated with the deficiency of the gene, but also for diseases such as cancer, where the administration of one or more genes may represent a less toxic and more efficient treatment than conventional therapies.

Another innovation to emerge from the human genome project is that most of the RNA generated does not code for proteins; rather, a significant proportion of RNA molecules have regulatory functions, such as microRNAs and long or non-coding RNA. Analysis of their role in different pathological states may lead to the identification of new therapeutic targets, the identification of biomarkers and the development of new experimental tools.

In addition, in recent years we have been able to confirm the role played by post-transcriptional modification processes in the regulation of gene expression and in protein functionality. Understanding these mechanisms and developing strategies to modulate them is a very promising strategy for the development of treatments for different diseases, as diabetes or cancer.

The goals of our Gene Therapy and Regulation of Gene Expression program are the following:

  • To develop efficient and safe gene-therapy vectors for the treatment of severe diseases. To achieve greater knowledge of the molecular mechanisms involved in the functionality of non-coding RNA and protein modifications, with the final objective of identifying new therapeutic targets and new treatments.

The final underlying objective is the development of new therapeutic strategies for diseases that lack treatment or for which treatment is ineffective.

Our research team is primarily basic but with a very translational vision for projects and we work closely with teams at the Clínica Universidad de Navarra, especially with the Departments of Hepatology and Oncology. As part of the cross-sectional program at CIMA, the program's researchers also collaborate closely with the other CIMA programs.



"Our laboratory is working on developing new treatments for liver diseases, based on gene transfer using one of the safest and most effective viral vectors currently in existence: the adeno-associated virus. In the next few years, we hope to obtain a clear clinical benefit in patients with rare diseases such as hyperoxaluria", Dr. Cristian Smerdou, Program Director.


Cristina López
Avda. Pío XII, 55
31008 Pamplona

(+34) 948 194 700 Ext. 6021