What are the risks and side effects of gene transfer in DMD?
While the results from preclinical studies are promising, there may be risks associated with treatment, such as immune reactions to the viral vector or other side effects. It is not yet known how often side effects might occur or how severe, serious or long-lasting they may be. Clinical trials are needed to best evaluate both the safety and efficacy of gene transfer candidates in DMD.
Importantly, it is known that some patients may already have developed antibodies against AAV because it is a naturally-occurring virus. If their antibody levels are high, gene transfer using AAV may be unsafe and/or unlikely to work, so such patients are currently excluded from receiving AAV vectors in clinical trials. Researchers are currently working on solutions for this challenge.