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.
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