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Jayesh A. Kulkarni, Dominik Witzigmann, Sarah B. Thomson, Sam Chen, Blair R. Leavitt, Pieter R. Cullis, Roy van der Meel
The increasing number of approved nucleic acid therapeutics demonstrates the potential to treat diseases by targeting their genetic blueprints in vivo. Conventional treatments generally induce therapeutic effects that are transient because they target proteins rather than underlying causes. In contrast, nucleic acid therapeutics can achieve long-lasting or even curative effects via gene inhibition, addition, replacement or editing. Their clinical translation, however, depends on delivery technologies that improve stability, facilitate internalization and increase target affinity. Here, we review four platform technologies that have enabled the clinical translation of nucleic acid therapeutics: antisense oligonucleotides, ligand-modified small interfering RNA conjugates, lipid nanoparticles and adeno-associated virus vectors. For each platform, we discuss the current state-of-the-art clinical approaches, explain the rationale behind its development, highlight technological aspects that facilitated clinical translation and provide an example of a clinically relevant genetic drug. In addition, we discuss how these technologies enable the development of cutting-edge genetic drugs, such as tissue-specific nucleic acid bioconjugates, messenger RNA and gene-editing therapeutics. This Review provides an overview of four platform technologies that are currently used in the clinic for delivery of nucleic acid therapeutics, describing their properties, discussing technical advancements that led to clinical approval, and highlighting examples of approved genetic drugs that make use of these technologies.
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Kulkarni, J. A., Witzigmann, D., Thomson, S. B., Chen, S., Leavitt, B. R., Cullis, P. R., & Meel, R. V. D. (2021). The current landscape of nucleic acid therapeutics. Nature Nanotechnology, 16(6), 630–643. https://doi.org/10.1038/s41565-021-00898-0
Kulkarni, Jayesh A., et al. "The current landscape of nucleic acid therapeutics." Nature Nanotechnology, vol. 16, no. 6, 2021, pp. 630–643, https://doi.org/10.1038/s41565-021-00898-0.
Kulkarni, Jayesh A., Dominik Witzigmann, Sarah B. Thomson, Sam Chen, Blair R. Leavitt, Pieter R. Cullis, and Roy van der Meel. "The current landscape of nucleic acid therapeutics." Nature Nanotechnology 16, no. 6 (2021): 630–643. https://doi.org/10.1038/s41565-021-00898-0.
Kulkarni J. A., Witzigmann D., Thomson S. B. [и др.] The current landscape of nucleic acid therapeutics // Nature Nanotechnology. — 2021. — Vol. 16, № 6. — P. 630–643. — URL: https://doi.org/10.1038/s41565-021-00898-0
@article{kulkarni2021current,
title = {{The current landscape of nucleic acid therapeutics}},
author = {Jayesh A. Kulkarni and Dominik Witzigmann and Sarah B. Thomson and Sam Chen and Blair R. Leavitt and Pieter R. Cullis and Roy van der Meel},
journal = {Nature Nanotechnology},
year = {2021},
volume = {16},
number = {6},
pages = {630--643},
doi = {10.1038/s41565-021-00898-0},
url = {https://doi.org/10.1038/s41565-021-00898-0}
}TY - JOUR AU - Kulkarni, Jayesh A. AU - Witzigmann, Dominik AU - Thomson, Sarah B. AU - Chen, Sam AU - Leavitt, Blair R. AU - Cullis, Pieter R. AU - Meel, Roy van der TI - The current landscape of nucleic acid therapeutics JO - Nature Nanotechnology VL - 16 IS - 6 SP - 630 EP - 643 PY - 2021 DA - 2021/05/31 DO - 10.1038/s41565-021-00898-0 UR - https://doi.org/10.1038/s41565-021-00898-0 ER -
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