Adeno - associated viruses (AAVs) make
fantastic gene delivery vehicles for episomal gene expression and are particularly useful for gene delivery to the nervous system.
Wilson had studied whether a harmless gene therapy tool called adeno - associated virus (AAV) can serve as
a gene delivery vehicle to treat inherited diseases like cystic fibrosis and hemophilia.
There is a size limit in terms of what you can package into
the gene delivery vehicle, and otoferlin is too large.