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Jote Bulcha, Yi Wang, Hong Ma, Phillip W.L. Tai, Guangping Gao
Throughout its 40-year history, the field of gene therapy has been marked by many transitions. It has seen great strides in combating human disease, has given hope to patients and families with limited treatment options, but has also been subject to many setbacks. Treatment of patients with this class of investigational drugs has resulted in severe adverse effects and, even in rare cases, death. At the heart of this dichotomous field are the viral-based vectors, the delivery vehicles that have allowed researchers and clinicians to develop powerful drug platforms, and have radically changed the face of medicine. Within the past 5 years, the gene therapy field has seen a wave of drugs based on viral vectors that have gained regulatory approval that come in a variety of designs and purposes. These modalities range from vector-based cancer therapies, to treating monogenic diseases with life-altering outcomes. At present, the three key vector strategies are based on adenoviruses, adeno-associated viruses, and lentiviruses. They have led the way in preclinical and clinical successes in the past two decades. However, despite these successes, many challenges still limit these approaches from attaining their full potential. To review the viral vector-based gene therapy landscape, we focus on these three highly regarded vector platforms and describe mechanisms of action and their roles in treating human disease.
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Bulcha, J., Wang, Y., Ma, H., Tai, P. W., & Gao, G. (2021). Viral vector platforms within the gene therapy landscape. Signal Transduction and Targeted Therapy, 6(1), 53. https://doi.org/10.1038/s41392-021-00487-6
Bulcha, Jote, et al. "Viral vector platforms within the gene therapy landscape." Signal Transduction and Targeted Therapy, vol. 6, no. 1, 2021, pp. 53, https://doi.org/10.1038/s41392-021-00487-6.
Bulcha, Jote, Yi Wang, Hong Ma, Phillip W.L. Tai, and Guangping Gao. "Viral vector platforms within the gene therapy landscape." Signal Transduction and Targeted Therapy 6, no. 1 (2021): 53. https://doi.org/10.1038/s41392-021-00487-6.
Bulcha J., Wang Y., Ma H. [и др.] Viral vector platforms within the gene therapy landscape // Signal Transduction and Targeted Therapy. — 2021. — Vol. 6, № 1. — P. 53. — URL: https://doi.org/10.1038/s41392-021-00487-6
@article{bulcha2021viral,
title = {{Viral vector platforms within the gene therapy landscape}},
author = {Jote Bulcha and Yi Wang and Hong Ma and Phillip W.L. Tai and Guangping Gao},
journal = {Signal Transduction and Targeted Therapy},
year = {2021},
volume = {6},
number = {1},
pages = {53},
doi = {10.1038/s41392-021-00487-6},
url = {https://doi.org/10.1038/s41392-021-00487-6}
}TY - JOUR AU - Bulcha, Jote AU - Wang, Yi AU - Ma, Hong AU - Tai, Phillip W.L. AU - Gao, Guangping TI - Viral vector platforms within the gene therapy landscape JO - Signal Transduction and Targeted Therapy VL - 6 IS - 1 SP - 53 PY - 2021 DA - 2021/02/08 DO - 10.1038/s41392-021-00487-6 UR - https://doi.org/10.1038/s41392-021-00487-6 ER -
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