CyTA - Journal of Food, Journal Year: 2025, Volume and Issue: 23(1)
Published: Jan. 21, 2025
Language: Английский
Journal of the American Chemical Society, Journal Year: 2023, Volume and Issue: 145(14), P. 7677 - 7691
Published: March 29, 2023
Functional nucleic acid (NA)-based drugs have a broad range of applications since they allow the alteration and control gene/protein expression patterns in cells. In principle, functional NAs need to be transported precisely efficiently target cells guarantee both functionality safety. Owing their negative charges, it is difficult for natural cross cell membrane composed lipid bilayer enter targeted Worse still, delivery undirected nontargeted healthy and/or tissues would induce unpredictable adverse effects. Therefore, specific cells/organs, particularly extrahepatic sites, required. Since aptamers can bind various proteins on surface with high specificity selectivity, serve as molecular recognition units accurately subsequently enable efficient cargo. this perspective, we summarize original, proof-of-concept aptamer-based strategies NAs. A few examples are then discussed, followed by our perspectives some challenges opportunities that lie ahead.
Language: Английский
Citations
63
Pharmaceutics, Journal Year: 2023, Volume and Issue: 15(1), P. 178 - 178
Published: Jan. 4, 2023
Gene therapy, as an emerging therapeutic approach, has shown remarkable advantages in the treatment of some major diseases. With deepening genomics research, people have gradually realized that emergence and development many diseases are related to genetic abnormalities. Therefore, nucleic acid drugs becoming a new boon (especially tumors diseases). It is conservatively estimated global market will exceed $20 billion by 2025. They simple design, mature synthesis, good biocompatibility. However, shortcomings acid, such poor stability, low bioavailability, targeting, greatly limit clinical application acid. Liposome nanoparticles can wrap internal cavities, increase stability prolong blood circulation time, thus improving transfection efficiency. This review focuses on recent advances potential applications liposome modified with (DNA, RNA, ASO) different chemical molecules (peptides, polymers, dendrimers, fluorescent molecules, magnetic nanoparticles, receptor targeting molecules). The ability deliver also discussed detail. We hope this help researchers design safer more efficient accelerate gene therapy.
Language: Английский
Citations
38
Ageing Research Reviews, Journal Year: 2024, Volume and Issue: 95, P. 102229 - 102229
Published: Feb. 15, 2024
Language: Английский
Citations
16
Signal Transduction and Targeted Therapy, Journal Year: 2024, Volume and Issue: 9(1)
Published: Nov. 29, 2024
Language: Английский
Citations
14
Life Sciences, Journal Year: 2024, Volume and Issue: 344, P. 122576 - 122576
Published: March 14, 2024
Language: Английский
Citations
9
Biomaterials Research, Journal Year: 2022, Volume and Issue: 26(1)
Published: Sept. 30, 2022
Antisense oligonucleotides (ASOs) are an important tool for the treatment of many genetic disorders. However, similar to other gene drugs, vectors often required protect them from degradation and clearance, accomplish their transport in vivo. Compared with viral vectors, artificial nonviral nanoparticles have a variety design, synthesis, formulation possibilities that can be selected protection delivery specific applications, they served critical therapeutic purposes animal model research clinical allowing safe efficient processes into target cells. We believe as new ASO drugs develop, exploration corresponding is inevitable. Intensive development improved strategies based on targets continue expand value approaches. Here, we provide overview current strategies, including ASOs modifications, action mechanisms, multi-carrier methods, which aim address irreplaceable role progressive delivery.
Language: Английский
Citations
30
International Journal of Molecular Sciences, Journal Year: 2023, Volume and Issue: 24(1), P. 746 - 746
Published: Jan. 1, 2023
RNA-mediated drugs are a rapidly growing class of therapeutics. Over the last five years, list FDA-approved RNA therapeutics has expanded owing to their unique targets and prolonged pharmacological effects. Their absorption, distribution, metabolism, excretion (ADME) have important clinical im-plications, but pharmacokinetic properties not been fully understood. Most structural modifications prevent rapid elimination from plasma administered intravenously or subcutaneously, with some exceptions, for effective distribution target organs. Distribution into tissues depends on addition moiety that can be transported show low volume because molecular size negatively-charged backbone. Nucleases metabolize shortened chain, metabolic ratio is relatively low. Therefore, most excreted in intact form. This review covers only ADME features also pharmacology data such as drug–drug interaction population analyses. As market expected expand, comprehensive knowledge will contribute interpreting evaluating properties.
Language: Английский
Citations
22
The Science of The Total Environment, Journal Year: 2023, Volume and Issue: 877, P. 162951 - 162951
Published: March 21, 2023
Language: Английский
Citations
22
Pharmacological Research, Journal Year: 2023, Volume and Issue: 195, P. 106853 - 106853
Published: July 18, 2023
Organ-on-chip (OoC) technology has led to in vitro models with many new possibilities compared conventional and vivo models. In this review, the potential of OoC improve prediction human oral bioavailability intrinsic clearance is discussed, a focus on functionality application current drug development practice. Multi-OoC demonstrating for pharmacokinetic (PK) studies are summarized existing challenges identified. Physiological parameters minimal viable platform multi-OoC model study PK provided, together specific read-outs recommendations relevant reference compounds validate model. Finally, translation profiles which will be required routinely apply during development.
Language: Английский
Citations
18
Molecular Therapy — Nucleic Acids, Journal Year: 2023, Volume and Issue: 32, P. 773 - 793
Published: May 9, 2023
Antisense oligonucleotide (ASO) therapeutics are being investigated for a broad range of neurological diseases. While ASOs have been effective in the clinic, improving productive ASO internalization into target cells remains key area focus field. Here, we how delivery ASO-loaded lipid nanoparticles (LNPs) affects activity, subcellular trafficking, and distribution brain. We show that ASO-LNPs increase activity up to 100-fold cultured primary brain as compared non-encapsulated ASO. However, contrast widespread uptake observed following free
Language: Английский
Citations
17