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Translational Unit


The TIGEM Translational Unit (Ttu) was set-up in order to develop in vivo large-scale preclinical studies for the treatment of different inherited disorders by using standardized and optimized protocols to facilitate translation from the bench to the bedside.   It counts with dedicated scientific staff to  perform pre- clinical research to fill gap between discovery and a clinical trial

The Ttu provides a wide range of preclinical testing services:

  • in vivo administration of nanomolecules/compounds
  • in vivo and ex vivo gene therapy approaches

in wild type and inherited diseases animal models (small and large animal models) by using different administration routes: oral, intravenous, intraperitoneal, Intra CSF (intraventricular, lumbar, intra cisterna magna) subcutaneous and local administration. Services also include tissue collection, processing and analysis.  

The Ttu collaborates with both TIGEM investigators and/orwith external collaborators (academic and pharmaceutical companies) to develop experimental design, formulation, administration and assessment of any potential therapeutic compound including the interpretation of study results.

Approaches include:

Standardized experimental procedures; Assays set-up
Pre-clinical studies in diverse animal models, doses and timing
Planning and overview of IND-enabling non clinical studies

Other Services:

  • Animal house for small and large animals
  • Availability of different mouse models of LSDs (MSD, MPSs and others) 
  • Formulation and administration of compounds.
  • Production of viral vector in collaboration with internal TIGEM vector core facility.
  • Tissue collection
  • Biochemical and IHC analysis
  • Interpretation of results and statistics