, Volume 52, Issue 12, pp 1590-1599

Targeting strategies for adeno-associated viral vector

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Adeno-associated virus (AAV) has rapidly become a promising gene delivery vector for its excellent advantages of low pathogenicity and long-term gene expression over other viral vectors for human gene therapy. However, the disadvantage of AAV for clinical gene therapy is the broad host range and the lack of tissue or cell specificity, which makes AAVs relatively inefficient gene delivery to target cells and short of safety. Therefore, it is required to improve the targeting capability of recombinant adeno-associated virus (rAAV) for successful gene therapy. At present, the successful identification of various-serotype AAVs and the improvement of rAAV production technology make it feasible to explore new applications of rAAV vector in clinical gene therapy trials. Moreover, the current development in targeting strategy has brought a bright prospect to rAAV vectors. Many studies have demonstrated that targeting gene therapy based on rAAV vectors could be achieved through transcription targeted, conjugate targeted, receptor targeted, gene recombinant targeted strategies, etc. This report gives a comprehensive review of the recent developments of targeting strategies for rAAV vectors.