At a time of unprecedented development of AAV-based gene therapies in neuromuscular disease, and in view of the difficulties encountered during therapeutic trials and in a real-life setting, German researchers have reviewed possible ways of improving the coexistence of humans with AAV itself. Among the many avenues mentioned, one should note the benefit of:
- targeting organs that are more immunotolerant to the AAVs, such as the brain, the liver and the eyes;
- inducing immunotolerance using reprogrammed regulatory T cells or medicines;
- using, more broadly, immunosuppressants to prevent any undesirable immune reaction;
- getting rid of neutralising antibodies against AAV;
- improving AAV itself.
