San Raffaele Telethon Institute for Gene Therapy
Immune Core
The Immune Core supports pre-clinical investigations of gene and cell therapies focusing on immune related questions and sets up immunological analyses enabling immune-monitoring in clinical trials. Specifically, the Core has been recently implemented with the aims of i) supporting Research Groups to identify, characterize and overcome potential hurdles deriving from immune response togene therapy (GT) for the development of successful therapies (Immune-tolerance to GT); ii) developing immunological assays to monitor specific responses in GTclinical trials and supporting the design of immunological endpoints in GT clinical protocols; iii) contributing to the monitoring of immunogenicity of first-in-humantherapies by investigation of exploratory research endpoints.
Activities
The Immune Core contribute s to R&D studies by:
- characterizing immune response to transgene/vector components in GT pre-clinical studies (evaluati on of the feasibility and support for the experimental design of in vivo and in vitro studies);
- identifying new approaches to circumvent immune response in GT pre-clinical studies (immune-tolerance to GT);
- supporting/collaborating to research pre-clinical immunological studies within SR - Tiget;
- developing broadly applicable and ad hoc flow cytometry panels for analysis of lymphoid and myeloid compartments (mouse/human/NHP);
- supporting the generation of raw flow cytometry data suitable for high - dimensional data analysis in collaboration with the Bioinformatics Core;
- contributing to grant proposals, including scientific planning and pilot experiments.
The Immune Core provide s support to clinical studies by:
- support ing the design of immunological endpoints in GT clinical protocols;
- perform ing research endpoints in GT clinical protocols ( immunological studies for exploratory endpoints in patients enrolled in SR - Tiget clinical trials);
- developing immune assays for clinical endpoints (design of immune assays tailored to specific diseases, drafting of standard operating procedures, collaborati on with the SR - Tiget clinical lab and GLP test facility for assay transfer and validation) .
