Adeno - associated viruses (AAVs) make fantastic gene delivery vehicles for episomal gene expression and are particularly useful for gene delivery to the nervous system. (blog.addgene.org)
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. (sciencemag.org)
There is a size limit in terms of what you can package into the gene delivery vehicle, and otoferlin is too large. (sciencedaily.com)