Syngeneic AAV Pseudo-particles Potentiate Gene Transduction of AAV Vectors.

ABSTRACT: Adeno-associated virus (AAV) vectors have emerged as a safe and efficient gene therapy platform. One complication is that a significant amount of empty particles have always been generated as impurities during AAV vector production. However, the effects of such particles on AAV vector performance remain unclear. Here we systemically evaluated the biological properties of three types of "empty" AAV particles: syngeneic pseudo-vectors with partial AAV genomes derived from DNA of the corresponding full particles, allogeneic pseudo-vectors with partial genomes different from the corresponding full particles, and null pseudo-vectors with no DNA inside the capsids. The syngeneic particles in excess increased the corresponding full AAV vector transgene expression both in vivo and in vitro. Howeve

SUBMITTER: Wang Q 

PROVIDER: S-EPMC5363323 | biostudies-literature | 2017 Mar

REPOSITORIES: biostudies-literature