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Curr Opin Pharmacol. 2015 Oct;24:59-67. doi: 10.1016/j.coph.2015.07.006. Epub 2015 Aug 25.

Adeno-associated virus serotypes for gene therapeutics.

Author information

1
Gene Transfer, Targeting and Therapeutics Core, The Salk Institute for Biological Studies, 10010 North Torrey Pines Road, San Diego, CA, USA.
2
Gene Therapy Research Unit, The Children's Hospital at Westmead and Children's Medical Research Institute, Locked Bag 4001, Westmead 2145, NSW, Australia.
3
Gene Therapy Research Unit, The Children's Hospital at Westmead and Children's Medical Research Institute, Locked Bag 4001, Westmead 2145, NSW, Australia; Discipline of Paediatrics and Child Health, The University of Sydney, NSW, Australia. Electronic address: ian.alexander@health.nsw.gov.au.

Abstract

Gene transfer vectors based on adeno-associated virus (AAV) are showing exciting therapeutic promise in early phase clinical trials. The ability to cross-package the prototypic AAV2 vector genome into different capsids is a powerful way of conferring novel tropism and biology, with evolving capsid engineering technologies and directed evolution approaches further enhancing the utility and flexibility of these vectors. Novel properties of specific capsids show unpredictable species and cell-type specificity. Therefore, full realisation of the therapeutic potential of AAV vectors requires the development of more therapeutically predictive preclinical methods for evaluating capsid performance. This will strongly complement an iterative approach to the evaluation of capsid variants in the clinic and, should wherever possible, include the determination of gene transfer efficiencies.

PMID:
26291407
DOI:
10.1016/j.coph.2015.07.006
[Indexed for MEDLINE]

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