Fondazione Telethon (FTLELE.IGM)

Telethon

Partner description and expertise

Fondazione Telethon (TIGEM) is currently located in the upper town quarters of Pozzuoli, in Naples. The institute counts with 5000 square meters converted in laboratories, office and research services: 4 “open space" laboratories, 4 meeting rooms, over 28 offices for researchers and administrative staff, dedicated spaces to microscopy, cell culture, bioinformatics, and an auditorium capable of seating 180 people for business, training and scientific dissemination. The institute will provide ample laboratory, facilities and office space to allow the Applicant to conduct his research project, as well as full administrative support. TIGEM has hosted more than 50 students and fellows from foreign countries, and has coordinated or participated in more than 20 EU-based projects and Marie Curie Training Networks; it has collaboration agreements with the University of Naples “Federico II”, The “Second” University of Naples and the UK-based Open University.

Role in the BATCure project

TIGEM will mostly contribute to BATCure by focussing on the generation and development of cell-based HC imaging assays to screen target focus libraries of small molecules in relevant cellular models of Batten disease. In particular, this partner will be in charge of (1) validating, by industrial standards of quantitative biology, relevant read-outs and cellular models of Batten disease generated by the consortium to generate HC imaging assays in 384-well format; (2) screening target focus libraries and potential compound hits in primary and secondary assays; (3) generating leads to correct Batten disease phenotype with the consortium.

Mr. Diego L. Medina is the Head of Fondazione Telethon’s High Content Screening Facility. Mr. Medina’s expertise is mainly in the cell biology of treat Lysosomal Storage Disorders and in quantitative biology approaches to validate high through put assays. He is interested in the development of innovative therapeutical approaches to treat Lysosomal Storage Disorders by using state-of the art high content imaging approaches to identify small molecules/correctors of pathologic lysosomal accumulation, as well as identifying new druggable targets involved in the pathological pathways of these diseases.