CLINICAL TRIALS AND TRANSLATIONAL MEDICINE COMMENTARY: Drug Delivery Trends in Clinical Trials and Translational Medicine: Challenges and Opportunities in the Delivery of Nucleic Acid-Based Therapeutics
Open accessAbstract
The ability to deliver nucleic acids (e.g., plasmid DNA, antisense oligonucleotides, siRNA) offers the potential to develop potent vaccines and novel therapeutics. However, nucleic acid-based therapeutics are still in their early stages as a new category of biologics. The efficacy of nucleic acids requires that these molecules be delivered to the interior of the target cell, which greatly complicates delivery strategies and compromises efficiency. Due to the safety concerns of viral vectors, synthetic vectors such as liposomes and polymers are preferred for the delivery of nucleic acid-based therapeutics. Yet, delivery efficiencies of synthetic vectors in the clinic are still too low to obtain therapeutic levels of gene expression. In this review, we focus on some key issues in the field of nucleic acid delivery such as PEGylation, encapsulation and targeted delivery and provide some perspectives for consideration in the development of improved synthetic vectors.
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Cationic non-viral vectors used to condense negatively charged CRISPR RNPs and plasmids are prone to interact with plasma proteins and physiological salt, resulting in recognition and clearance by RES.
“On the other hand, cationic non-viral vectors used to condense negatively charged CRISPR RNPs and plasmids are prone to interact with plasma proteins and the physiological salt in physiological conditions, resulting in the recognition and clearance by the reticuloendothelial system (RES) [141–143].”
From Non-viral delivery systems for CRISPR/Cas9-based genome editing: challenges and opportunities (2018) · cites this paper as [143]
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- Authors
- Xu, L., & Anchordoquy, T.
- Affiliations
- University of Colorado Denver (US)
- Corresponding author
- Anchordoquy, Thomas
- Venue
- Journal of Pharmaceutical Sciences
- Publisher
- Elsevier BV
- Published
- 2011-01
- Volume
- 100
- Issue
- 1
- Pages
- 38-52
- ISSN
- 0022-3549 (ISSN-L)
- PMC
- PMC3303188
- OpenAlex
- W2090160156
- Cited by
- 209
- References
- 155
- Citations by year
- 2026: 8 · 2025: 9 · 2024: 9 · 2023: 12 · 2022: 7 · 2021: 6 · 2020: 9 · 2019: 9 · 2018: 9 · 2017: 14 · 2016: 13 · 2015: 14 · 2014: 15 · 2013: 28 · 2012: 28
- Type
- journal article
- Language
- en
- Open access
- bronze
- doi.org
- Locations
- Journal of Pharmaceutical Sciences (publishedVersion)
- PubMed (publishedVersion)
- PubMed Central (acceptedVersion)
- Funders
- National Institutes of Health
- National Institute of Biomedical Imaging and Bioengineering (R01EB005476)
- Topics
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › RNA Interference and Gene Delivery
- Physical Sciences › Materials Science › Biomaterials › Nanoparticle-Based Drug Delivery
- Life Sciences › Pharmacology, Toxicology and Pharmaceutics › Pharmaceutical Science › Advanced Drug Delivery Systems
- Keywords
- nucleic acid delivery, nucleic acid therapeutics, antisense oligonucleotides, siRNA, plasmid DNA, liposomes, polymeric vectors, PEGylation, encapsulation, targeted delivery, synthetic vectors, gene therapy, drug delivery
- MeSH terms
- Animals, Biological Therapy, Chemistry, Pharmaceutical, Humans, Nucleic Acids, Oligonucleotides, Plasmids, Oligonucleotides, Antisense, Drug Delivery Systems, Vaccines, DNA, RNA, Small Interfering
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