Cell therapy

This area of research aims to develop and optimize cell transplantation protocols in pre-clinical models of muscular dystrophy and connective tissue disorders It is based upon intra-arterial delivery of vessel-associated, pericyte-derived progenitors cells (termed mesoangioblasts) either wt of genetically corrected to express the therapeutic gene product.

Research areas

Current areas of interest are:

  • Dissecting the different step of transplantation to enhance efficacy
  • Developing and testing lentivectors expressing snRNA to induce exon skipping (for large genes) or the cDNA for small genes
  • Developing novel intra-arterial delivery approaches
  • Developing a long term organ culture of a functional muscle
  • Testing biomaterials for enhanced engraftment of cells for topic delivery