DMD04

Trial Name: Mesoangioblast-mediated exon skipping for genetic correction of exon 51 mutation, based upon a single injection in individual skeletal muscles of five non ambulant patients affected by Duchenne Muscular Dystrophy: a non-randomized, open label, phase I/IIa study

Chief Investigator: Dr Imelda Hughes

Principal Investigator: Professor Giulio Cossu

Trial Description: A Phase I/IIa, non-randomized, open label study

Background: There are currently limited treatments available for Duchenne Muscular Dystrophy patients. This study uses a lentiviral vector to transduce, differentiate and inject patient mesoangioblasts (MABS) to genetically correct exon 51, potentially inducing synthesis of dystrophin in the injected muscle.

Main Outcomes: To determine the safety of injected autologous MABS after genetic modification and their subsequent ability to engraft and induce dystrophin synthesis.

Patients to be recruited: 5

Status: Set-up

CTU: N/A (Manchester CRF)

Funder: Wellcome Trust

Further Information: Trial Manager Dr Rebecca Cole rebecca.cole@manchester.ac.uk