Emerging Delivery Systems for Enabling Precision Nucleic Acid Therapeutics
Nucleic acid therapeutics represent a highly promising treatment approach in modern medicine, treating diseases at the genetic level. However, these therapeutics face numerous challenges in practical applications, particularly regarding their stability, effectiveness, cellular uptake efficiency, and...
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| Published in | ACS nano Vol. 19; no. 4; pp. 4039 - 4083 |
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| Main Authors | , , , , , , , |
| Format | Journal Article |
| Language | English |
| Published |
United States
American Chemical Society
04.02.2025
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| Online Access | Get full text |
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| Abstract | Nucleic acid therapeutics represent a highly promising treatment approach in modern medicine, treating diseases at the genetic level. However, these therapeutics face numerous challenges in practical applications, particularly regarding their stability, effectiveness, cellular uptake efficiency, and limitations in delivering them specifically to target tissues. To overcome these obstacles, researchers have developed various innovative delivery systems, including viral vectors, lipid nanoparticles, polymer nanoparticles, inorganic nanoparticles, protein carriers, exosomes, antibody oligonucleotide conjugates, and DNA nanostructure-based delivery systems. These systems enhance the therapeutic efficacy of nucleic acid drugs by improving their stability, targeting specificity, and half-life in vivo. In this review, we systematically discuss different types of nucleic acid drugs, analyze the major barriers encountered in their delivery, and summarize the current research progress in emerging delivery systems. We also highlight the latest advancements in the application of these systems for treating genetic diseases, infectious diseases, cancer, brain diseases, and wound healing. This review aims to provide a comprehensive overview of nucleic acid drug delivery systems’ current status and future directions by integrating the latest advancements in nanotechnology, biomaterials science, and gene editing technologies, emphasizing their transformative potential in precision medicine. |
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| AbstractList | Nucleic acid therapeutics represent a highly promising treatment approach in modern medicine, treating diseases at the genetic level. However, these therapeutics face numerous challenges in practical applications, particularly regarding their stability, effectiveness, cellular uptake efficiency, and limitations in delivering them specifically to target tissues. To overcome these obstacles, researchers have developed various innovative delivery systems, including viral vectors, lipid nanoparticles, polymer nanoparticles, inorganic nanoparticles, protein carriers, exosomes, antibody oligonucleotide conjugates, and DNA nanostructure-based delivery systems. These systems enhance the therapeutic efficacy of nucleic acid drugs by improving their stability, targeting specificity, and half-life in vivo. In this review, we systematically discuss different types of nucleic acid drugs, analyze the major barriers encountered in their delivery, and summarize the current research progress in emerging delivery systems. We also highlight the latest advancements in the application of these systems for treating genetic diseases, infectious diseases, cancer, brain diseases, and wound healing. This review aims to provide a comprehensive overview of nucleic acid drug delivery systems’ current status and future directions by integrating the latest advancements in nanotechnology, biomaterials science, and gene editing technologies, emphasizing their transformative potential in precision medicine. Nucleic acid therapeutics represent a highly promising treatment approach in modern medicine, treating diseases at the genetic level. However, these therapeutics face numerous challenges in practical applications, particularly regarding their stability, effectiveness, cellular uptake efficiency, and limitations in delivering them specifically to target tissues. To overcome these obstacles, researchers have developed various innovative delivery systems, including viral vectors, lipid nanoparticles, polymer nanoparticles, inorganic nanoparticles, protein carriers, exosomes, antibody oligonucleotide conjugates, and DNA nanostructure-based delivery systems. These systems enhance the therapeutic efficacy of nucleic acid drugs by improving their stability, targeting specificity, and half-life in vivo. In this review, we systematically discuss different types of nucleic acid drugs, analyze the major barriers encountered in their delivery, and summarize the current research progress in emerging delivery systems. We also highlight the latest advancements in the application of these systems for treating genetic diseases, infectious diseases, cancer, brain diseases, and wound healing. This review aims to provide a comprehensive overview of nucleic acid drug delivery systems' current status and future directions by integrating the latest advancements in nanotechnology, biomaterials science, and gene editing technologies, emphasizing their transformative potential in precision medicine.Nucleic acid therapeutics represent a highly promising treatment approach in modern medicine, treating diseases at the genetic level. However, these therapeutics face numerous challenges in practical applications, particularly regarding their stability, effectiveness, cellular uptake efficiency, and limitations in delivering them specifically to target tissues. To overcome these obstacles, researchers have developed various innovative delivery systems, including viral vectors, lipid nanoparticles, polymer nanoparticles, inorganic nanoparticles, protein carriers, exosomes, antibody oligonucleotide conjugates, and DNA nanostructure-based delivery systems. These systems enhance the therapeutic efficacy of nucleic acid drugs by improving their stability, targeting specificity, and half-life in vivo. In this review, we systematically discuss different types of nucleic acid drugs, analyze the major barriers encountered in their delivery, and summarize the current research progress in emerging delivery systems. We also highlight the latest advancements in the application of these systems for treating genetic diseases, infectious diseases, cancer, brain diseases, and wound healing. This review aims to provide a comprehensive overview of nucleic acid drug delivery systems' current status and future directions by integrating the latest advancements in nanotechnology, biomaterials science, and gene editing technologies, emphasizing their transformative potential in precision medicine. |
| Author | Li, Fengyong Bian, Xiaochun Li, Haohao Zhou, Liping Wen, Yongqiang Luo, Zhiwei Hang, Zhongci Liu, Guotao |
| AuthorAffiliation | Beijing Key Laboratory for Bioengineering and Sensing Technology, Daxing Research Institute, School of Chemistry and Biological Engineering Plastic Surgery Hospital, Peking Union Medical College |
| AuthorAffiliation_xml | – name: Beijing Key Laboratory for Bioengineering and Sensing Technology, Daxing Research Institute, School of Chemistry and Biological Engineering – name: Plastic Surgery Hospital, Peking Union Medical College |
| Author_xml | – sequence: 1 givenname: Xiaochun surname: Bian fullname: Bian, Xiaochun organization: Beijing Key Laboratory for Bioengineering and Sensing Technology, Daxing Research Institute, School of Chemistry and Biological Engineering – sequence: 2 givenname: Liping orcidid: 0009-0001-9293-2766 surname: Zhou fullname: Zhou, Liping email: Liping-Zhou@ustb.edu.cn organization: Beijing Key Laboratory for Bioengineering and Sensing Technology, Daxing Research Institute, School of Chemistry and Biological Engineering – sequence: 3 givenname: Zhiwei surname: Luo fullname: Luo, Zhiwei organization: Beijing Key Laboratory for Bioengineering and Sensing Technology, Daxing Research Institute, School of Chemistry and Biological Engineering – sequence: 4 givenname: Guotao surname: Liu fullname: Liu, Guotao organization: Beijing Key Laboratory for Bioengineering and Sensing Technology, Daxing Research Institute, School of Chemistry and Biological Engineering – sequence: 5 givenname: Zhongci surname: Hang fullname: Hang, Zhongci organization: Beijing Key Laboratory for Bioengineering and Sensing Technology, Daxing Research Institute, School of Chemistry and Biological Engineering – sequence: 6 givenname: Haohao surname: Li fullname: Li, Haohao organization: Beijing Key Laboratory for Bioengineering and Sensing Technology, Daxing Research Institute, School of Chemistry and Biological Engineering – sequence: 7 givenname: Fengyong surname: Li fullname: Li, Fengyong email: lify202108@163.com organization: Plastic Surgery Hospital, Peking Union Medical College – sequence: 8 givenname: Yongqiang orcidid: 0000-0002-1924-4166 surname: Wen fullname: Wen, Yongqiang email: wyq_wen@ustb.edu.cn organization: Beijing Key Laboratory for Bioengineering and Sensing Technology, Daxing Research Institute, School of Chemistry and Biological Engineering |
| BackLink | https://www.ncbi.nlm.nih.gov/pubmed/39834294$$D View this record in MEDLINE/PubMed |
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| CitedBy_id | crossref_primary_10_3390_ijms26062491 crossref_primary_10_1016_j_ijbiomac_2025_142136 |
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| Snippet | Nucleic acid therapeutics represent a highly promising treatment approach in modern medicine, treating diseases at the genetic level. However, these... |
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| StartPage | 4039 |
| SubjectTerms | Animals Drug Carriers - chemistry Drug Delivery Systems Humans Nanoparticles - chemistry Nucleic Acids - administration & dosage Nucleic Acids - chemistry Nucleic Acids - therapeutic use Precision Medicine |
| Title | Emerging Delivery Systems for Enabling Precision Nucleic Acid Therapeutics |
| URI | http://dx.doi.org/10.1021/acsnano.4c11858 https://www.ncbi.nlm.nih.gov/pubmed/39834294 https://www.proquest.com/docview/3157554788 |
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