Combating immunity

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...

Non-viral delivery

Non-viral delivery for gene therapy A next generation gene therapy delivery approach The Duchenne Research Fund has committed £640,000 to accelerate research into alternative ways to deliver gene therapy to Duchenne patients, with the aim of increasing the number of...

CRISPR

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...

GOSH coordinator

Great Ormond Street Hospital Clinical Trial Co-ordinator Manju Agarwal As part of the Newcastle Plan in 2015/16, eight patient organisations contributed £1.2m to fund at least 16 vital posts across the UK to support and expand clinical trial capacity for Duchenne...

Gene therapy for Becker

Gene therapy for Becker A microdystrophin gene therapy approach for Becker muscular dystrophy We have awarded a grant to the team of Professor Thomas Voit (pictured) at the Great Ormond Street Biomedical Research Centre at UCL to develop a microdystrophin gene therapy...