Four Ways to Package Transgenes That Exceed the Size Limit of Adeno-associated Virus
Type
Blog Post
Published
Sept. 1, 2020, 1:15 p.m.
...and drawbacks of four split AAV vector approaches.
1. Overlapping
Overview: The overlapping strategy has...Adeno-Associated Virus Capsids. Human Gene Therapy Methods 27:1–12 . https://doi.org/10.1089/hgtb.2015.140
Frischmeyer...that the oversized AAV genomes are fragmented or truncated when packaged and then following transduction,...and may need to be optimized for each unique application (Trapani et al., 2015).
Expression of smaller...transgene to help prevent the accumulation of such truncated proteins.
Examples of use:
Ghosh et al., 2008...design, donor A may be capable of expressing a truncated protein. This can be avoided by excluding a stop...JS (2011) Gene Therapy of mdx Mice With Large Truncated Dystrophins Generated by Recombination Using rAAV6...