Novel genetic approaches for DMD: addressing immune response challenges

Joanna Pomeroy, PhD Candidate, University of Portsmouth, Portsmouth, UK, discusses the potential of novel genetic approaches for the treatment of Duchenne muscular dystrophy (DMD). The use of gene therapy approaches in DMD presents both promise and controversy, as numerous challenges associated with targeting the DMD gene has been identified. As DMD is caused by a loss of full-length dystrophin protein, one might assume that replacing the dystrophin protein or restoring the function of the DMD gene could potentially lead to a cure for the disease. However, it is important to note that while some individuals with DMD still possess shorter isoforms of the protein, others have no isoforms at all. Introducing the DMD gene as a genetic approach therefore has the potential to elicit an immune response. This detrimental immune response has been observed in various trials, and it becomes a critical factor to consider when evaluating gene therapies. If optimized, however, these genetic approaches hold promise for improving both muscle wastage and cognitive impairments in individuals with DMD. This interview took place at The BNA 2023 International Festival of Neuroscience in Brighton, UK.

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