Mesoangioblast-mediated exon skipping

Mesoangioblast-mediated exon skipping for genetic correction of exon 51 mutation

Giulio Cossu
University of Manchester, UK

Tuesday 24th October 2017

This is a proposal from Professor Giulio Cossu and colleagues at Manchester University to develop mesoangioblast-mediated exon skipping for genetic correction of exon 51 mutation as a therapy for Duchenne muscular dystrophy. The proposal aims to use mesangioblasts (MABs) treated ex vivo with a virally-mediated strategy to exclude exon 51 into the final DMD transcript. The study will focus on safety and measuring functional improvement in patients’ thumb muscles.

The committee valued the previous experience of the investigator and the accomplished team associated with this proposal. They also welcomed the wealth of data from a previous clinical trial.

The report makes recommendations and suggestions on the development of important additional pre-clinical data, and for the clinical trial, the benefit of accompanying vascular imaging, precision in dystrophin quantification by multiple analytic methods, and on the use of double-blinded analysis of muscles injected with cells or only physiological solution.

To request a full copy of the report please contact the applicant Giulio Cossu

20 Dec 2017