Renal Gene Therapy and Chimeric AAV Development

"About 15% of the population in developed and developing countries has chronic kidney disease (CKD). Between 10-15% is associated with a genetic alteration (70% in pediatrics). Developing gene therapy vectors targeting renal cells will allow us to offer them treatments."

DR. RAFAEL ALDABE ARREGUI
RESEARCHER. RENAL GENE THERAPY AND CHIMERIC AAV DEVELOPMENT RESEARCH GROUP

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Renal Gene Therapy and Chimeric AAV Development

This research group is dedicated to developing gene therapy treatments for genetic diseases affecting kidney cells. First, we are identifying adeno-associated viruses (AAVs) that efficiently transduce renal cells. We utilize AAV libraries, obtained through genetic modifications of the capsid, to select AAV variants with renal tropism in both mice and non-human primates.

Additionally, we are exploring the potential of creating chimeric AAVs to provide them with new properties, such as increased potency and evasion of immune system responses. We are also designing AAVs with highly specific tropisms. Our strategy is based on substituting AAV9 capsid residues with reactive non-natural amino acids to chemically modify the AAV surface. This allows us to attach glycans, aptamers, and other molecules using "click chemistry" reactions.

The primary kidney disease we are focusing on is autosomal dominant polycystic kidney disease.

Dr. Rafael Aldabe

GROUP LEADER

	+34 948 194 700 \| Ext. 81 4030
	raldabe@unav.es
	Research profile

Objectives of our research

Gene therapy vectors

Develop gene therapy vectors based on adeno-associated viruses capable of transducing various types of renal cells.

Binding to the AAV

Study the effect of binding different molecules to the AAV capsid to optimize properties such as tropism and potency.

Create therapies

Create therapies to treat genetic renal diseases, starting with autosomal dominant polycystic kidney disease, using the developed renal vectors.

Priority diseases in our research group

Autosomal dominant polycystic kidney disease

The first renal disease we are addressing is autosomal dominant polycystic kidney disease. Mutations in one of two genes, PKD1 or PKD2, account for approximately 78% and 15% of ADPKD cases. Between 6-10% of renal patients undergoing replacement therapy have polycystic kidney disease.