Incorporation of aptamers in the terminal loop of shRNAs yields an effective and novel combinatorial targeting strategy.

ABSTRACT: Gene therapy by engineering patient's own blood cells to confer HIV resistance can potentially lead to a functional cure for AIDS. Toward this goal, we have previously developed an anti-HIV lentivirus vector that deploys a combination of shRNA, ribozyme and RNA decoy. To further improve this therapeutic vector against viral escape, we sought an additional reagent to target HIV integrase. Here, we report the development of a new strategy for selection and expression of aptamer for gene therapy. We developed a SELEX protocol (multi-tag SELEX) for selecting RNA aptamers against proteins with low solubility or stability, such as integrase. More importantly, we expressed these aptamers in vivo by incorporating them in the terminal loop of shRNAs. This novel strategy allowed efficient expression

SUBMITTER: Pang KM 

PROVIDER: S-EPMC5758892 | biostudies-literature | 2018 Jan

REPOSITORIES: biostudies-literature