| abstract |
The present disclosure provides methods of expressing a transgene in a cell, methods of treating a disorder in a subject in need thereof, and pharmaceutical compositions. In particular, the method comprises treating a cell (eg, a cell of a subject suffering from a disorder such as cystic fibrosis) into a recombinant adeno-associated virus (rAAV) comprising, in one embodiment, a polynucleotide comprising an AV.TL65 capsid protein and a transgene. ) in combination with an enhancer of AAV transduction, thereby expressing the transgene in the cell. The present disclosure also provides in one embodiment a pharmaceutical composition comprising a rAAV comprising a polynucleotide comprising an AV.TL65 capsid protein and a transgene in combination with one or more enhancers. |