Genome editing using CRISPR-Cas9 methodology mediated by an adenovirus-associated virus (AAV) has shown its effectiveness in mice but comes up against the impossibility of repeating injections of the viral vector. Japanese researchers have successfully overcome the obstacle of anti-AAV neutralizing antibodies by using a non-viral vector:
- lipid nanoparticles with low immunogenic power make it possible to transport the CRISPR-Cas9 system containing messenger RNAs,
- the functional result (measured by skipping exon 45 of the dystrophin gene) is very respectable, although transient,
- repeated injections have proven to be feasible and not harmful, as have loco-regional infusions to target several muscle groups.