Combating immunity to AAV Ensuring as many patients as possible can benefit from gene therapy The Duchenne Research Fund has awarded a grant to Dr Barry Byrne and Dr Manuela Corti’s team at the University of Florida, whose vital work focuses on overcoming...
CRISPR for Duchenne Genome editing technology to correct the mutated dystrophin gene The Duchenne Research Fund has awarded a £200,000 grant to the team at the Hospital for Sick Children in Toronto, Canada (‘Sick Kids’), who are working on using...
Gene therapy for Becker A microdystrophin gene therapy approach for Becker muscular dystrophy The Duchenne Research Fund has awarded a grant to the team of Professor Thomas Voit (pictured) at the Great Ormond Street Biomedical Research Centre at UCL to develop a...
LTBP4 Targeting muscle inflammation and fibrosis With the funds raised in 2016, the DRF is tackling inflammation and fibrosis, which are two key problems for Duchenne boys. It is believed that the protein LTBP4 prevents the activation of a key biological pathway...
