Research Project

Project Title:

National Center for Gene Therapy and Drugs based on RNA Technology

Project Type:

Translational research

Disease group(s):

Hereditary glomerulopathies

Project Summary:

The main purpose of this project is to develop and validate an innovative gene editing (GE) approach for Alport Syndrome targeting the podocytes, key affected cells of the disease and unique producers of collagen in the kidney. We will test and validate the technology in-vitro directly on these relevant cells, isolated non-invasively from human urine samples. In parallel we will work on the translationality of this approach, targeting specific COL4A5 variants in dog and mouse models, starting from in-vitro editing on fibroblasts. Once editing will be achieved in-vitro we will move on in-vivo preclinical infection using AAV9.

Lead principal investigator(s):

Rosario Rizzuto, Siena


Alessandra Renieri, Siena
Anna Maria Pinto, Siena
Sergio Daga, Siena

Project Period:

01/2022   -   10/2025



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