The DOI matches its Crossref record; 8 of the 8 references it cites were found in a registry or an index.
Displaying High-affinity Ligands on Adeno-associated Viral Vectors Enables Tumor Cell-specific and Safe Gene Transfer
Open accessAbstract
Gene transfer vectors derived from the adeno-associated virus (AAV) have recently received increasing attention due to substantial therapeutic benefit in several clinical trials. Nevertheless, their great potential for in vivo gene therapy can only be partially exploited owing to their broad tropism. Current cell surface targeting strategies expanded vector tropism towards transduction of cell types that are inefficiently infected naturally, but failed to restrict or fully re-direct AAV's tropism. Hypothesizing that this limitation can be overcome by equipping natural receptor-blinded AAV vectors with high-affinity ligands, we displayed designed ankyrin repeat proteins (DARPin) as VP2 fusion proteins on AAV capsids ablated for natural primary receptor binding. These second generation targeting vectors demonstrated an as of yet unachieved efficiency to discriminate between target and non-target cells in mono- and mixed cultures. Moreover, DARPin-AAV vectors delivered a suicide gene precisely to tumor tissue and substantially reduced tumor growth without causing fatal liver toxicity. The latter caused death in animals treated with conventional AAV vectors with unmodified capsids, which accumulated in liver tissue and failed to affect tumor growth. This novel targeting platform will be key to translational approaches requiring restricted and cell type-specific in vivo gene delivery.
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Therapeutic in vivo gene transfer for genetic disease using AAV: Progress and challenges
Mingozzi, F., & High, K. A. (2011) · Nature Reviews Genetics · doi:10.1038/nrg2988 · Cited by 893
AAV vector integration sites in mouse hepatocellular carcinoma
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High-affinity binders selected from designed ankyrin repeat protein libraries
Binz, H. K., Amstutz, P., Kohl, A., Stumpp, M. T., Briand, C., Forrer, P., Grütter, M. G., & Plückthun, A. (2004) · Nature Biotechnology · doi:10.1038/nbt962 · Cited by 656
Identification of a heparin-binding motif on adeno-associated virus type 2 capsids
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Nathwani, A. C., Rosales, C., McIntosh, J., Rastegarlari, G., Nathwani, D., Raj, D., Nawathe, S., Waddington, S. N., Bronson, R., Jackson, S., et al. (2011) · Molecular Therapy · doi:10.1038/mt.2010.274 · Cited by 345
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Cited by
Adeno-Associated Virus (AAV) as a Vector for Gene Therapy
Naso, M. F., Tomkowicz, B., Perry, W. L., & Strohl, W. R. (2017) · BioDrugs · doi:10.1007/s40259-017-0234-5 · Cited by 1,362
Adeno-associated virus as a delivery vector for gene therapy of human diseases
Wang, J.-H., Gessler, D. J., Zhan, W., Gallagher, T. L., & Gao, G. (2024) · Signal Transduction and Targeted Therapy · doi:10.1038/s41392-024-01780-w · Cited by 904
Engineering adeno-associated viruses for clinical gene therapy
Kotterman, M. A., & Schaffer, D. V. (2014) · Nature Reviews Genetics · doi:10.1038/nrg3742 · Cited by 761
Capsid modifications for targeting and improving the efficacy of AAV vectors
Büning, H., & Srivastava, A. (2019) · Molecular Therapy - Methods & Clinical Development · doi:10.1016/j.omtm.2019.01.008 · Cited by 260
Self-citationDavidsson, M., Wang, G., Aldrin-Kirk, P., Cardoso, T., Nolbrant, S., Hartnor, M., Mudannayake, J., Parmar, M., & Björklund, T. (2019) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.1910061116 · Cited by 142
Metadata
- Authors
- Münch, R. C., Janicki, H., Völker, I., Rasbach, A., Hallek, M., Büning, H., & Buchholz, C. J.
- Venue
- Molecular Therapy
- Publisher
- Elsevier BV
- Published
- 2013-01
- Volume
- 21
- Issue
- 1
- Pages
- 109-118
- ISSN
- 1525-0016 (ISSN-L)
- OpenAlex
- W1973733861
- Cited by
- 179
- References
- 8
- FWCI
- 4.96
- Type
- journal article
- Language
- en
- Open access
- hybrid
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- Münch, R. C., Janicki, H., Völker, I., Rasbach, A., Hallek, M., Büning, H., & Buchholz, C. J. (2013). Displaying High-affinity Ligands on Adeno-associated Viral Vectors Enables Tumor Cell-specific and Safe Gene Transfer. Molecular Therapy. https://doi.org/10.1038/mt.2012.186
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