The DOI matches its Crossref record; 44 of the 44 references it cites were found in a registry or an index.
Gene therapy with helper-dependent adenoviral vectors: lessons from studies in large animal models
Abstract
Helper-dependent adenoviral vectors (HDAd) are deleted of all viral genes and they can efficiently transduce a wide variety of dividing and non-dividing cells to mediate high transgene expression levels. Unlike early generation adenoviral vectors, the absence of viral genes in HDAd results in long-term transgene expression without chronic toxicity and permits a large cloning capacity of 36 kb. Moreover, HDAd genomes exist extra-chromosomally thus minimizing the risks of germline transmission and insertional mutagenesis. For these reasons, HDAd offers tremendous potential for in vivo gene therapy. This chapter reviews preclinical studies using HDAd in large animal models to assess safety and efficacy in a wide variety of gene therapy applications.
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“Maximum serum concentration of genes in gene augmentation therapy gradually decline to about 10% in 24 months due to acute phase immune responses and neutralizing antibodies, thus hampering the efficacy of gene augmentation therapy [34].”
From Viral Delivery Systems for CRISPR (2019) · cites this paper as [34]
Citations
Cites
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Brunetti-Pierri, N., Palmer, D. J., Beaudet, A. L., Carey, K. D., Finegold, M., & Ng, P. (2004) · Human Gene Therapy · doi:10.1089/10430340460732445 · Cited by 256
Self-citationMok, H., Palmer, D. J., Ng, P., & Barry, M. A. (2005) · Molecular Therapy · doi:10.1016/j.ymthe.2004.09.015 · Cited by 232
Self-citationLiver transplantation for glycogen storage disease types I, III, and IV
Matern, D., Starzl, T. E., Arnaout, W., Barnard, J., Bynon, J. S., Dhawan, A., Emond, J., Haagsma, E. B., Hug, G., Lachaux, A., et al. (1999) · European Journal of Pediatrics · doi:10.1007/pl00014320 · Cited by 185
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Episomal Persistence of Recombinant Adenoviral Vector Genomes during the Cell Cycle In Vivo
Ehrhardt, A., Xu, H., & Kay, M. A. (2003) · Journal of Virology · doi:10.1128/jvi.77.13.7689-7695.2003 · Cited by 93
Brunetti‐Pierri, N., Stapleton, G. E., Law, M. A., Breinholt, J. P., Palmer, D. J., Zuo, Y., Grove, N. C., Finegold, M. J., Rice, K., Beaudet, A. L., et al. (2008) · Molecular Therapy · doi:10.1038/mt.2008.257 · Cited by 89
Self-citation
Cited by
Viral Delivery Systems for CRISPR
Xu, C. L., Ruan, M. Z. C., Mahajan, V. B., & Tsang, S. H. (2019) · Viruses · doi:10.3390/v11010028 · Cited by 277
Viral Vectors for Gene Transfer
Chen, Y. H., Keiser, M. S., & Davidson, B. L. (2018) · Current Protocols in Mouse Biology · doi:10.1002/cpmo.58 · Cited by 146
Metadata
- Authors
- Brunetti-Pierri, N., & Ng, P.
- Venue
- Virus Genes
- Published
- 2017-06-07
- Volume
- 53
- Issue
- 5
- Pages
- 684-691
- ISSN
- 0920-8569
- 1572-994X
- Cited by
- 32
- References
- 44
- Language
- en
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- Brunetti-Pierri, N., & Ng, P. (2017). Gene therapy with helper-dependent adenoviral vectors: lessons from studies in large animal models. Virus Genes. https://doi.org/10.1007/s11262-017-1471-x
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Bibliographic data: Semantic Scholar, OpenAlex, Crossref.
Contains information from the Semantic Scholar Academic Graph Datasets, made available under the ODC Attribution License (ODC-BY 1.0).
Semantic Scholar’s data is described in: Kinney, R., et al. (2023). The Semantic Scholar Open Data Platform. arXiv:2301.10140.
