Site Specific Integration of Recombinant AAV Vector Genomes by Rep68 Protein Expressed on the Surface of AAV Particles

Adeno-associated virus (AAV) vectors are replication defective viruses that are engineered to deliver therapeutic genetic cargo to cells. The structural and enzymatic AAV proteins are traditionally supplied “in trans” to generate engineered particles for gene delivery. One constraint of AAV vectors is the size limitation of the genetic insert. Gene therapy experts at Nationwide Children’s Hospital have engineered an AAV vector that expresses the Rep78 protein on the surface of the viral particle thus eliminating the need to package the rep coding region within the particle. This strategy allows for delivery of a functional Rep78 protein while increasing the overall therapeutic gene insert size.

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