Gene Therapy and Regulation of Gene Expression
"Thanks to gene therapy, we expect to obtain in the coming years a clear clinical benefit in patients with rare diseases."
DR. CRISTIAN SMERDOU PICAZO DIRECTOR. GENE THERAPY AND REGULATION OF GENE EXPRESSION RESEARCH PROGRAM
The Gene Therapy and Regulation of Gene Expression Program at Cima aims to develop new therapeutic strategies for diseases that lack treatment or whose treatment is unsatisfactory.
We investigate to improve therapies developed for genetic diseases and cancer in order to reach their clinical translation, the implementation of new gene therapy technologies such as gene editing, the use of non-viral vectors to transfer DNA or RNA in vivo, or the development of new expression regulation systems, as well as the identification of new therapeutic targets based on lncRNAs or UPR-related processes.
The research team is fundamentally basic, but with a very translational vision and maintains close collaborations with teams from the Clínica Universidad de Navarra. In addition, the Gene Therapy and Regulation of Gene Expression program is a transversal program that collaborates very actively with the other programs of the Cima Universidad de Navarra.
Currently, the Program has numerous national and international research projects, actively participates in European calls and maintains collaborations with biotechnology companies that represent a unique opportunity to continue advancing.
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Gene Therapy Research
Program Objectives
Development of new therapeutic strategies based on gene transfer for inherited and acquired diseases for which there is no cure or for which current therapy is not satisfactory.
Analysis of the function and mechanisms of action of long non-coding RNAs in the development of pathologies and their application in the development of new therapies.
Analysis of the role of UPR and post-translational modifications of proteins in the regulation of gene expression and their implication in the development of diseases and new therapies.
Cima gene therapy clinical trial
Our translational vocation moves us to investigate therapeutic alternatives that reach patients. This is the case of VTX-801, a drug originated at Cima for the treatment of Wilson's disease, with technology licensed to the biotechnology company Vivet Therapeutics.
Currently, the pharmaceutical company Pfizer Inc. and Vivet Therapeutics have the GATEWAY study underway, a Phase 1/2 clinical trial evaluating the efficacy of the TX-801 vector for the treatment of patients affected by this rare disease.
Our research groups
We seek to improve therapeutic alternatives for currently incurable diseases
Gene Therapy of Liver Diseases
Gene Therapy of Congenital Diseases with Neurological Affectation
Gene therapy of renal diseases and study of N-terminal acetylation of proteins
Long Non-Coding RNAs and Cancer Regulation
Gene Therapy for Cancer
Non-Coding RNAs in Hepatocarcinoma and Other Liver Diseases
Endoplasmic Reticulum Stress Response in Neurodegenerative Diseases
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.
Would you like to help us?
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.
Meet the research team
Scientific activity of the Gene Therapy and
and Regulation of Gene Expression Program
Latest scientific publications
- Use of an AAV serotype Anc80 to provide durable cure of phenylketonuria in a mouse model Nov 1, 2021 | Magazine: Journal of Inherited Metabolic Disease
- Endogenous retroelement activation by epigenetic therapy reverses the Warburg effect and elicits mitochondrial-mediated cancer cell death May 1, 2021 | Magazine: Cancer Discovery
- AAV-HDV: An Attractive Platform for the In Vivo Study of HDV Biology and the Mechanism of Disease Pathogenesis Apr 28, 2021 | Magazine: Viruses
- Adenovirus-Mediated Inducible Expression of a PD-L1 Blocking Antibody in Combination with Macrophage Depletion Improves Survival in a Mouse Model of Peritoneal Carcinomatosis Apr 17, 2021 | Magazine: International Journal of Molecular Sciences