Adeno-associated virus (AAV) represent one of the most efficient gene therapy vehicles. However, its limited cargo capacity has hampered its use for conditions that require delivery of DNA sequences that exceed 5 kb in size, including not only the transgene but also the cis regulatory elements that are necessary for its expression.

Splittera split inteins in gene therapy:

– overcome gene size limitations of viral vectors

– induce in vivo covalent reconstitution of large full-length proteins expressed by different viral vectors

– compatible with CRISPR/Cas9 genome editing nuclease or genes related to inherited and non-inherited diseases

Splittera split inteins can also be used to increase the specificity of viral particles for a particular cell tissue by allowing the insertion of large binding proteins into different regions of the viral capsid which act as targeting molecules.