期刊
ADVANCED DRUG DELIVERY REVIEWS
卷 156, 期 -, 页码 119-132出版社
ELSEVIER
DOI: 10.1016/j.addr.2020.06.014
关键词
Gene delivery; Gene editing; Polymeric vehicle; Nanocarriers; Polyplex; Nanoparticle; Biodegradable; Biocompatible; Sustained release; In vivo
资金
- National Institutes of Health (NIH) [UG3 HL147352, R01 HL125892]
- NIH National Research Service Awards (NRSAs) [T32 GM86287]
- NIH NRSA training grant [T32 DK101019, T32 DK007276]
- [F32 HL142144]
Polymeric vehicles are versatile tools for therapeutic gene delivery. Many polymers-when assembled with nucleic acids into vehicles-can protect the cargo from degradation and clearance in vivo, and facilitate its transport into intracellular compartments. Design options in polymer synthesis yield a comprehensive range of molecules and resulting vehicle formulations. These properties can be manipulated to achieve stronger association with nucleic acid cargo and cells, improved endosomal escape, or sustained delivery depending on the application. Here, we describe current approaches for polymer use and related strategies for gene delivery in preclinical and clinical applications. Polymer vehicles delivering genetic material have already achieved significant therapeutic endpoints in vitro and in animal models. From our perspective, with preclincal assays that better mimic the in vivo environment, improved strategies for target specificity, and scalable techniques for polymer synthesis, the impact of this therapeutic approach will continue to expand. (C) 2020 Elsevier B.V. All rights reserved.
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