CRISPR

CRISPR for Duchenne Genome editing technology to correct the mutated dystrophin gene Duchenne muscular dystrophy is caused by a mutation in the dystrophin gene, which results in the absence of full-length dystrophin protein, needed to protect muscles. Without this...

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...