Centre Hospitalier Universitaire Vaudois (Switzerland) – www.chuv.ch
SCIDNET Principal Investigator: Fabio Candotti (Fabio.Candotti@chuv.ch)
For the past 16 years, Dr. Candotti has led a research group in the field of gene therapy that has performed preclinical development of a series of viral vectors for the correction of several primary immunodeficiencies (PIDs) including SCIDX-1, JAK3-SCID, ADA-SCID, IL12Rb1-deficiency, and the Wiskott-Aldrich syndrome (WAS). In addition, Dr. Candotti has co-developed a series of 3 gene therapy trials for ADA-SCID using retroviral vectors and contributed to the development of an ongoing trial of lentiviral gene therapy for the same disease. He has published ~30 articles in the area of gene therapy for PIDs. Dr. Candotti’s team research team has recently applied zinc finger nuclease and CRISPR/Cas-9 technologies to target the WAS locus in mice (Mol Ther 2014 22, S216-S217) using approaches similar to the ones proposed in the grant application. Technical/scientific added value that the CHUV will bring to the proposed project as a partner. In addition to the experience in gene transfer, Dr. Candotti’s group has extensive expertise in the cellular and biochemical aspects of ADA deficiency that will be required for the execution of the proposed tasks.