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Articles de revues sur le sujet « Nucleic acid based drug »

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Auteur : Grafiati
Publié le 11 mars 2023

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1

Tan, Xuyu, Fei Jia, Ping Wang et Ke Zhang. « Nucleic acid-based drug delivery strategies ». Journal of Controlled Release 323 (juillet 2020) : 240–52. http://dx.doi.org/10.1016/j.jconrel.2020.03.040.

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Kim, Haejoo, et Minseok Kwak. « Structures and Applications of Nucleic Acid-Based Micelles for Cancer Therapy ». International Journal of Molecular Sciences 24, no 2 (13 janvier 2023) : 1592. http://dx.doi.org/10.3390/ijms24021592.

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Nucleic acids have become important building blocks in nanotechnology over the last 30 years. DNA and RNA can sequentially build specific nanostructures, resulting in versatile drug delivery systems. Self-assembling amphiphilic nucleic acids, composed of hydrophilic and hydrophobic segments to form micelle structures, have the potential for cancer therapeutics due to their ability to encapsulate hydrophobic agents into their core and position functional groups on the surface. Moreover, DNA or RNA within bio-compatible micelles can function as drugs by themselves. This review introduces and discusses nucleic acid-based spherical micelles from diverse amphiphilic nucleic acids and their applications in cancer therapy.
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Kuriyama, Naoya, Yusuke Yoshioka, Shinsuke Kikuchi, Akihiko Okamura, Nobuyoshi Azuma et Takahiro Ochiya. « Challenges for the Development of Extracellular Vesicle-Based Nucleic Acid Medicines ». Cancers 13, no 23 (6 décembre 2021) : 6137. http://dx.doi.org/10.3390/cancers13236137.

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Nucleic acid drugs, such as siRNAs, antisense oligonucleotides, and miRNAs, exert their therapeutic effects by causing genetic changes in cells. However, there are various limitations in their delivery to target organs and cells, making their application to cancer treatment difficult. Extracellular vesicles (EVs) are lipid bilayer particles that are released from most cells, are stable in the blood, and have low immunogenicity. Methods using EVs to deliver nucleic acid drugs to target organs are rapidly being developed that take advantage of these properties. There are two main methods for loading nucleic acid drugs into EVs. One is to genetically engineer the parent cell and load the target gene into the EV, and the other is to isolate EVs and then load them with the nucleic acid drug. Target organ delivery methods include passive targeting using the enhanced permeation and retention effect of EVs and active targeting in which EVs are modified with antibodies, peptides, or aptamers to enhance their accumulation in tumors. In this review, we summarize the advantages of EVs as a drug delivery system for nucleic acid drugs, the methods of loading nucleic acid drugs into EVs, and the targeting of EVs to target organs.
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Wu, Yuanbing, Ania Rashidpour, María Pilar Almajano et Isidoro Metón. « Chitosan-Based Drug Delivery System : Applications in Fish Biotechnology ». Polymers 12, no 5 (21 mai 2020) : 1177. http://dx.doi.org/10.3390/polym12051177.

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Chitosan is increasingly used for safe nucleic acid delivery in gene therapy studies, due to well-known properties such as bioadhesion, low toxicity, biodegradability and biocompatibility. Furthermore, chitosan derivatization can be easily performed to improve the solubility and stability of chitosan–nucleic acid polyplexes, and enhance efficient target cell drug delivery, cell uptake, intracellular endosomal escape, unpacking and nuclear import of expression plasmids. As in other fields, chitosan is a promising drug delivery vector with great potential for the fish farming industry. This review highlights state-of-the-art assays using chitosan-based methodologies for delivering nucleic acids into cells, and focuses attention on recent advances in chitosan-mediated gene delivery for fish biotechnology applications. The efficiency of chitosan for gene therapy studies in fish biotechnology is discussed in fields such as fish vaccination against bacterial and viral infection, control of gonadal development and gene overexpression and silencing for overcoming metabolic limitations, such as dependence on protein-rich diets and the low glucose tolerance of farmed fish. Finally, challenges and perspectives on the future developments of chitosan-based gene delivery in fish are also discussed.
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de Vries, Jan Willem, Feng Zhang et Andreas Herrmann. « Drug delivery systems based on nucleic acid nanostructures ». Journal of Controlled Release 172, no 2 (décembre 2013) : 467–83. http://dx.doi.org/10.1016/j.jconrel.2013.05.022.

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Mulvey, Matthew C., Margaret Lemmon, Stephanie Rotter, Jonathan Lees, Leo Einck et Carol A. Nacy. « Optimization of a Nucleic Acid-Based Reporter System To Detect Mycobacterium tuberculosis Antibiotic Sensitivity ». Antimicrobial Agents and Chemotherapy 59, no 1 (3 novembre 2014) : 407–13. http://dx.doi.org/10.1128/aac.03135-14.

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ABSTRACTWe previously reported the development of a prototype antibiotic sensitivity assay to detect drug-resistantMycobacterium tuberculosisusing infection by mycobacteriophage to create a novel nucleic acid transcript, a surrogate marker of mycobacterial viability, detected by reverse transcriptase PCR (M. C. Mulvey et al., mBio3:e00312-11, 2012). This assay detects antibiotic resistance to all drugs, even drugs for which the resistance mechanism is unknown or complex: it is a phenotypic readout using nucleic acid detection. In this report, we describe development and characteristics of an optimized reporter system that directed expression of the RNA cyclase ribozyme, which generated circular RNA through an intramolecular splicing reaction and led to accumulation of a new nucleic acid sequence in phage-infected bacteria. These modifications simplified the assay, increased the limit of detection from 104to <102M. tuberculosiscells, and correctly identified the susceptibility profile ofM. tuberculosisstrains exposed for 16 h to either first-line or second-line antitubercular drugs. In addition to phenotypic drug resistance or susceptibility, the assay reported streptomycin MICs and clearly detected 10% drug-resistant cells in an otherwise drug-susceptible population.
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Ashrafuzzaman, Md. « Aptamers as Both Drugs and Drug-Carriers ». BioMed Research International 2014 (2014) : 1–21. http://dx.doi.org/10.1155/2014/697923.

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Aptamers are short nucleic acid oligos. They may serve as both drugs and drug-carriers. Their use as diagnostic tools is also evident. They can be generated using various experimental, theoretical, and computational techniques. The systematic evolution of ligands by exponential enrichment which uses iterative screening of nucleic acid libraries is a popular experimental technique. Theory inspired methodology entropy-based seed-and-grow strategy that designs aptamer templates to bind specifically to targets is another one. Aptamers are predicted to be highly useful in producing general drugs and theranostic drugs occasionally for certain diseases like cancer, Alzheimer’s disease, and so on. They bind to various targets like lipids, nucleic acids, proteins, small organic compounds, and even entire organisms. Aptamers may also serve as drug-carriers or nanoparticles helping drugs to get released in specific target regions. Due to better target specific physical binding properties aptamers cause less off-target toxicity effects. Therefore, search for aptamer based drugs, drug-carriers, and even diagnostic tools is expanding fast. The biophysical properties in relation to the target specific binding phenomena of aptamers, energetics behind the aptamer transport of drugs, and the consequent biological implications will be discussed. This review will open up avenues leading to novel drug discovery and drug delivery.
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Pozharov, Vitaly P., et Tamara Minko. « Nanotechnology-Based RNA Vaccines : Fundamentals, Advantages and Challenges ». Pharmaceutics 15, no 1 (5 janvier 2023) : 194. http://dx.doi.org/10.3390/pharmaceutics15010194.

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Over the past decades, many drugs based on the use of nanotechnology and nucleic acids have been developed. However, until recently, most of them remained at the stage of pre-clinical development and testing and did not find their way to the clinic. In our opinion, the main reason for this situation lies in the enormous complexity of the development and industrial production of such formulations leading to their high cost. The development of nanotechnology-based drugs requires the participation of scientists from many and completely different specialties including Pharmaceutical Sciences, Medicine, Engineering, Drug Delivery, Chemistry, Molecular Biology, Physiology and so on. Nevertheless, emergence of coronavirus and new vaccines based on nanotechnology has shown the high efficiency of this approach. Effective development of vaccines based on the use of nucleic acids and nanomedicine requires an understanding of a wide range of principles including mechanisms of immune responses, nucleic acid functions, nanotechnology and vaccinations. In this regard, the purpose of the current review is to recall the basic principles of the work of the immune system, vaccination, nanotechnology and drug delivery in terms of the development and production of vaccines based on both nanotechnology and the use of nucleic acids.
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Campuzano, Susana, María Pedrero et José M. Pingarrón. « Electrochemical Nucleic Acid-Based Biosensing of Drugs of Abuse and Pharmaceuticals ». Current Medicinal Chemistry 25, no 33 (24 octobre 2018) : 4102–18. http://dx.doi.org/10.2174/0929867324666171121103156.

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Background: Studies on the interactions of DNA with small molecular drugs are currently performed both to explore their mechanism of action and to develop new drugs. Electrochemical biosensors offer a very promising alternative to more complex conventional techniques for drug determination due to rapidness, low cost, simplicity, high sensitivity and compatibility with use in different settings. In this review, selected electrochemical nucleic acid-based biosensing methods described so far for the determination of pharmaceuticals and illicit drugs are briefly overviewed, discussing their basics and main features. A section pointing out general conclusions and future directions in this field is also provided. Results: The 42 selected contributions described electrochemical platforms to determine drugs of interest by monitoring their specific interactions with nucleic acids (DNA and aptamers), DNA damage and specific DNA-protein interactions. The highlighted approaches reported the use of electrodes unmodified or modified with nanomaterials and/or polymers in which DNA-drug interaction was followed by electrochemical detection of DNA puric bases, active drug or diffusion-free markers, and monitoring changes in the surface layer morphology/permeability and charge transfer resistance using different electrochemical techniques. Conclusion: Although electrochemical nucleic acid biosensing approaches constitute an interesting option for drugs determination in terms of cost, simplicity and miniaturized instrumentation, validating exhaustively their performance in real samples against conventional methodologies and implementing them into portable and automatic high throughput devices, together with exploring novel electrode modifications with nanomaterials and polymers and studying in more detail their multiplexing ability for analysis of a large number of analytes, is still needed.
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Du, Rong, Chen Wang, Ling Zhu et Yanlian Yang. « Extracellular Vesicles as Delivery Vehicles for Therapeutic Nucleic Acids in Cancer Gene Therapy : Progress and Challenges ». Pharmaceutics 14, no 10 (19 octobre 2022) : 2236. http://dx.doi.org/10.3390/pharmaceutics14102236.

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Extracellular vesicles (EVs) are nanoscale vesicles secreted by most types of cells as natural vehicles to transfer molecular information between cells. Due to their low toxicity and high biocompatibility, EVs have attracted increasing attention as drug delivery systems. Many studies have demonstrated that EV-loaded nucleic acids, including RNA-based nucleic acid drugs and CRISPR/Cas gene-editing systems, can alter gene expressions and functions of recipient cells for cancer gene therapy. Here in this review, we discuss the advantages and challenges of EV-based nucleic acid delivery systems in cancer therapy. We summarize the techniques and methods to increase EV yield, enhance nucleic acid loading efficiency, extend circulation time, and improve targeted delivery, as well as their applications in gene therapy and combination with other cancer therapies. Finally, we discuss the current status, challenges, and prospects of EVs as a therapeutic tool for the clinical application of nucleic acid drugs.
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Sun, Lun Quan, et Jonathan P. Wong. « Frontiers in nucleic acid-based drug research and development ». Future Medicinal Chemistry 7, no 13 (septembre 2015) : 1619–21. http://dx.doi.org/10.4155/fmc.15.117.

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Booth, David E., et Kidong Lee. « Robust regression-based analysis of drug–nucleic acid binding ». Analytical Biochemistry 319, no 2 (août 2003) : 258–62. http://dx.doi.org/10.1016/s0003-2697(03)00290-2.

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Koo, Bonwoo, Haneul Yoo, Ho Jeong Choi, Min Kim, Cheoljae Kim et Ki Tae Kim. « Visible Light Photochemical Reactions for Nucleic Acid-Based Technologies ». Molecules 26, no 3 (21 janvier 2021) : 556. http://dx.doi.org/10.3390/molecules26030556.

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The expanding scope of chemical reactions applied to nucleic acids has diversified the design of nucleic acid-based technologies that are essential to medicinal chemistry and chemical biology. Among chemical reactions, visible light photochemical reaction is considered a promising tool that can be used for the manipulations of nucleic acids owing to its advantages, such as mild reaction conditions and ease of the reaction process. Of late, inspired by the development of visible light-absorbing molecules and photocatalysts, visible light-driven photochemical reactions have been used to conduct various molecular manipulations, such as the cleavage or ligation of nucleic acids and other molecules as well as the synthesis of functional molecules. In this review, we describe the recent developments (from 2010) in visible light photochemical reactions involving nucleic acids and their applications in the design of nucleic acid-based technologies including DNA photocleaving, DNA photoligation, nucleic acid sensors, the release of functional molecules, and DNA-encoded libraries.
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Sun, Yue, Lingxian Meng, Yuxin Zhang, Dan Zhao et Yunfeng Lin. « The Application of Nucleic Acids and Nucleic Acid Materials in Antimicrobial Research ». Current Stem Cell Research & ; Therapy 16, no 1 (1 décembre 2021) : 66–73. http://dx.doi.org/10.2174/1574888x15666200521084417.

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Due to the misuse of antibiotics, multiple drug-resistant pathogenic bacteria have increasingly emerged. This has increased the difficulty of treatment as these bacteria directly affect public health by diminishing the potency of existing antibiotics. Developing alternative therapeutic strategies is the urgent need to reduce the mortality and morbidity related to drug-resistant bacterial infections. In the past 10 to 20 years, nanomedicines have been widely studied and applied as an antibacterial agent. They have become a novel tool for fighting resistant bacteria. The most common innovative substances, metal and metal oxide nanoparticles (NPs), have been widely reported. Until recently, DNA nanostructures were used alone or functionalized with specific DNA sequences by many scholars for antimicrobial purposes which were alternatively selected as therapy for severe bacterial infections. These are a potential candidate for treatments and have a considerable role in killing antibiotic-resistant bacteria. This review involves the dimensions of multidrug resistance and the mechanism of bacteria developing drug resistance. The importance of this article is that we summarized the current study of nano-materials based on nucleic acids in antimicrobial use. Meanwhile, the current progress and the present obstacles for their antibacterial and therapeutic use and special function of stem cells in this field are also discussed.
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Cerrato, Carmine Pasquale, Tõnis Lehto et Ülo Langel. « Peptide-based vectors : recent developments ». Biomolecular Concepts 5, no 6 (1 décembre 2014) : 479–88. http://dx.doi.org/10.1515/bmc-2014-0024.

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AbstractPeptides and peptide-cargo complexes have been used for drug delivery and gene therapy. One of the most used delivery vectors are cell-penetrating peptides, due to their ability to be taken up by a variety of cell types and deliver a large variety of cargoes through the cell membrane with low cytotoxicity. In vitro and in vivo studies have shown their possibility and full effectiveness to deliver oligonucleotides, plasmid DNA, small interfering RNAs, antibodies, and drugs. We report in this review some of the latest strategies for peptide-mediated delivery of nucleic acids. It focuses on peptide-based vectors for therapeutic molecules and on nucleic acid delivery. In addition, we discuss recent applications and clinical trials.
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Gautherot, Isabelle, et Reg??s Sodoyer. « A Multi-Model Approach to Nucleic Acid-Based Drug Development ». BioDrugs 18, no 1 (2004) : 37–50. http://dx.doi.org/10.2165/00063030-200418010-00004.

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Muralidhara, Bilikallahalli K., Rinku Baid, Steve M. Bishop, Min Huang, Wei Wang et Sandeep Nema. « Critical considerations for developing nucleic acid macromolecule based drug products ». Drug Discovery Today 21, no 3 (mars 2016) : 430–44. http://dx.doi.org/10.1016/j.drudis.2015.11.012.

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Buhmann, Raymund, Ting Yang, Monica Schifferer, Martin Obermeier, Gundula Jaeger et Hans-Jochem Kolb. « Therapeutic Nucleic Acids : A Potential Source of Resistance to Cancer, Antiviral and Immunosuppressive Therapy. » Blood 112, no 11 (16 novembre 2008) : 1614. http://dx.doi.org/10.1182/blood.v112.11.1614.1614.

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Abstract According to their unique structural and chemical properties nucleic acids were recognized to provide inherent therapeutic potential beyond sole information storage. In the meantime an increasing number of nucleic acid based drugs achieved FDA approval and entered clinical trails (e.g. “antisense” or “immunosustimulatory CpG” oligodeoxynucletides (ODNs), aptamers, ribozymes, RNA interference or defibrotide). But so far, no or less information is available whether these compounds might compete with chemically and structurally related drugs, e.g. nucleoside analogues (NA) widely used in cancer or antiviral therapy, or interfere with the intracellular nucleic acid metabolism. In the present report we provide evidence, that nucleic-acid based drugs antagonize fludarabine, acyclovir or mycophenolate mofetil (MMF). In presence of defibrotide (DF), a polydisperse mixture of single-stranded oligodeoxyribonucleotides (15 to 30 kD) e.g. used for treatment of hepatic veno-occlusive disease and other endothelial disorders, fludarabine treated lymphocytes or myeloid blasts where rescued from apoptosis. According to nucleic acid degradation the resulting metabolite deoxycytidine turned out to be the key substrate competing with fludarabine for phosphorylation by deoxycitidine kinase (dCK) and suggested interference with nucleic acid metabolism rather than direct competition with the drug for cellular uptake. Moreover, in standard drug resistance assays using acyclovir sensitive herpes simplex virus (HSV) strains (V0631508), 4 mM of DF restored viral replication in presence of 50 mM acyclovir. This was confirmed by quantitative PCR of viral DNA. Here, deoxythymidine turned out to be the main competitor for intracellular phosphorylation mediated by virus thymidine kinase. To further extent our findings, that an increase of the extracellular concentration of nucleic acids directly interfere with the intracellular nucleic acid metabolism, mixed lymphocyte reactions (MLRs) were performed, to test whether the immunosuppressive effects of mycophenolate mofetil (MMF) could be reversed. As control cyclosporine A (CsA) was used. Here, addition of singular ribonucleotides almost completely antagonized the T cell inhibitory effects of mycophenolic acid (MPA), respectively its prodrug MMF, but not of CsA. As MPA is known to be a potent, selective, uncompetitive and reversible inhibitor of inosine monophosphate dehydrogenase, a key enzyme for the de novo pathway of guanosine nucleotide synthesis, addition of guanosine to the MLRs was found to be effective and sufficient to reverse the immunosuppressive effects of MPA. We conclude that treatment with nucleic-acid-based drugs interfere with the intracellular nucleic acid metabolism, thus decreasing the efficacy of NAs used for cancer and antiviral therapy or the immunosuppressive therapy using MMF. Prospective clinical trials are required to confirm these in vitro findings.
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Buhmann, Raymund, Ting Yang, Martin Obermeier, Gundula Jaeger et Hans-Jochem Kolb. « Exogenous Nucleic Acids - A Potential Source of Resistance to Nucleoside Analogues in Cancer and Antiviral Therapy. » Blood 110, no 11 (16 novembre 2007) : 4207. http://dx.doi.org/10.1182/blood.v110.11.4207.4207.

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Abstract The metabolism of exogenous nucleic acids is not well defined. Moreover, there is little information whether there might be interference with chemically and structurally related drugs, e.g. nucleoside analogues (NA) widely used in cancer or in antiviral therapy. In the present report we provide evidence, that nucleic-acid based drugs might antagonize fludarabine or acyclovir. In vitro, fludarabine treated lymphocytes or myeloid blasts where rescued from apoptosis when incubated with defibrotide (DF), a polydisperse mixture of single-stranded oligodeoxyribonucleotides (15 to 30 kD) or singular deoxynucleotides. Thereby deoxycytidine (dCTP) turned out to be the key substrate competing with fludarabine for phosphorylation by deoxycitidine kinase (dCK) and suggested interference with nucleic acid metabolism rather than direct competition with the drug. To further prove this hypothesis the influence of defibrotide on HSV replication was evaluated. In standard drug resistance assays performed with acyclovir sensitive herpes simplex virus (HSV) strains (V0631508) 4 mM of DF restored viral replication in presence of 50 mM acyclovir. This was confirmed by quantitative PCR of viral DNA. Moreover, in this case deoxythymidine (dTTP) turned out to be the competitor for intracellular phosphorylation mediated by virus thymidine kinase. We conclude that treatment with DF and other nucleic-acid-based drugs interfere with the efficacy of NA used for cancer and antiviral therapy. Prospective clinical trials are required to confirm these in vitro findings.
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Krüger, Arne, Flávia Zimbres, Thales Kronenberger et Carsten Wrenger. « Molecular Modeling Applied to Nucleic Acid-Based Molecule Development ». Biomolecules 8, no 3 (27 août 2018) : 83. http://dx.doi.org/10.3390/biom8030083.

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Molecular modeling by means of docking and molecular dynamics (MD) has become an integral part of early drug discovery projects, enabling the screening and enrichment of large libraries of small molecules. In the past decades, special emphasis was drawn to nucleic acid (NA)-based molecules in the fields of therapy, diagnosis, and drug delivery. Research has increased dramatically with the advent of the SELEX (systematic evolution of ligands by exponential enrichment) technique, which results in single-stranded DNA or RNA sequences that bind with high affinity and specificity to their targets. Herein, we discuss the role and contribution of docking and MD to the development and optimization of new nucleic acid-based molecules. This review focuses on the different approaches currently available for molecular modeling applied to NA interaction with proteins. We discuss topics ranging from structure prediction to docking and MD, highlighting their main advantages and limitations and the influence of flexibility on their calculations.
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Santosh, Baby, et Pramod K. Yadava. « Nucleic Acid Aptamers : Research Tools in Disease Diagnostics and Therapeutics ». BioMed Research International 2014 (2014) : 1–13. http://dx.doi.org/10.1155/2014/540451.

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Aptamers are short sequences of nucleic acid (DNA or RNA) or peptide molecules which adopt a conformation and bind cognate ligands with high affinity and specificity in a manner akin to antibody-antigen interactions. It has been globally acknowledged that aptamers promise a plethora of diagnostic and therapeutic applications. Although use of nucleic acid aptamers as targeted therapeutics or mediators of targeted drug delivery is a relatively new avenue of research, one aptamer-based drug “Macugen” is FDA approved and a series of aptamer-based drugs are in clinical pipelines. The present review discusses the aspects of design, unique properties, applications, and development of different aptamers to aid in cancer diagnosis, prevention, and/or treatment under defined conditions.
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Dantsu, Yuliya, Ying Zhang et Wen Zhang. « Advances in Therapeutic L-Nucleosides and L-Nucleic Acids with Unusual Handedness ». Genes 13, no 1 (24 décembre 2021) : 46. http://dx.doi.org/10.3390/genes13010046.

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Nucleic-acid-based small molecule and oligonucleotide therapies are attractive topics due to their potential for effective target of disease-related modules and specific control of disease gene expression. As the non-naturally occurring biomolecules, modified DNA/RNA nucleoside and oligonucleotide analogues composed of L-(deoxy)riboses, have been designed and applied as innovative therapeutics with superior plasma stability, weakened cytotoxicity, and inexistent immunogenicity. Although all the chiral centers in the backbone are mirror converted from the natural D-nucleic acids, L-nucleic acids are equipped with the same nucleobases (A, G, C and U or T), which are critical to maintain the programmability and form adaptable tertiary structures for target binding. The types of L-nucleic acid drugs are increasingly varied, from chemically modified nucleoside analogues that interact with pathogenic polymerases to nanoparticles containing hundreds of repeating L-nucleotides that circulate durably in vivo. This article mainly reviews three different aspects of L-nucleic acid therapies, including pharmacological L-nucleosides, Spiegelmers as specific target-binding aptamers, and L-nanostructures as effective drug-delivery devices.
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Lamberti, Gaetano, et Anna Angela Barba. « Drug Delivery of siRNA Therapeutics ». Pharmaceutics 12, no 2 (20 février 2020) : 178. http://dx.doi.org/10.3390/pharmaceutics12020178.

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Dailey, Magdalena M., Chayanendu Hait, Patrick A. Holt, Jon M. Maguire, Jason B. Meier, M. Clarke Miller, Luigi Petraccone et John O. Trent. « Structure-based drug design : From nucleic acid to membrane protein targets ». Experimental and Molecular Pathology 86, no 3 (juin 2009) : 141–50. http://dx.doi.org/10.1016/j.yexmp.2009.01.011.

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Epstein, David, Charles Wilson et Marty Stanton. « Archemix : drug discovery innovation based on evolutionary nucleic acid technology platforms ». Drug Discovery Today 7, no 5 (mars 2002) : S8—S11. http://dx.doi.org/10.1016/s1359-6446(02)02170-0.

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Della Pelle, Giulia, et Nina Kostevšek. « Nucleic Acid Delivery with Red-Blood-Cell-Based Carriers ». International Journal of Molecular Sciences 22, no 10 (17 mai 2021) : 5264. http://dx.doi.org/10.3390/ijms22105264.

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Gene therapy has the potential to become a staple of 21st-century medicine. However, to overcome the limitations of existing gene-delivery therapies, that is, poor stability and inefficient and delivery and accumulation of nucleic acids (NAs), safe drug-delivery systems (DDSs) allowing the prolonged circulation and expression of the administered genes in vivo are needed. In this review article, the development of DDSs over the past 70 years is briefly described. Since synthetic DDSs can be recognized and eliminated as foreign substances by the immune system, new approaches must be found. Using the body’s own cells as DDSs is a unique and exciting strategy and can be used in a completely new way to overcome the critical limitations of existing drug-delivery approaches. Among the different circulatory cells, red blood cells (RBCs) are the most abundant and thus can be isolated in sufficiently large quantities to decrease the complexity and cost of the treatment compared to other cell-based carriers. Therefore, in the second part, this article describes 70 years of research on the development of RBCs as DDSs, covering the most important RBC properties and loading methods. In the third part, it focuses on RBCs as the NA delivery system with advantages and drawbacks discussed to decide whether they are suitable for NA delivery in vivo.
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Hager, Simone, Frederic Julien Fittler, Ernst Wagner et Matthias Bros. « Nucleic Acid-Based Approaches for Tumor Therapy ». Cells 9, no 9 (9 septembre 2020) : 2061. http://dx.doi.org/10.3390/cells9092061.

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Within the last decade, the introduction of checkpoint inhibitors proposed to boost the patients’ anti-tumor immune response has proven the efficacy of immunotherapeutic approaches for tumor therapy. Furthermore, especially in the context of the development of biocompatible, cell type targeting nano-carriers, nucleic acid-based drugs aimed to initiate and to enhance anti-tumor responses have come of age. This review intends to provide a comprehensive overview of the current state of the therapeutic use of nucleic acids for cancer treatment on various levels, comprising (i) mRNA and DNA-based vaccines to be expressed by antigen presenting cells evoking sustained anti-tumor T cell responses, (ii) molecular adjuvants, (iii) strategies to inhibit/reprogram tumor-induced regulatory immune cells e.g., by RNA interference (RNAi), (iv) genetically tailored T cells and natural killer cells to directly recognize tumor antigens, and (v) killing of tumor cells, and reprograming of constituents of the tumor microenvironment by gene transfer and RNAi. Aside from further improvements of individual nucleic acid-based drugs, the major perspective for successful cancer therapy will be combination treatments employing conventional regimens as well as immunotherapeutics like checkpoint inhibitors and nucleic acid-based drugs, each acting on several levels to adequately counter-act tumor immune evasion.
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Giammona, Gaetano, et Emanuela Fabiola Craparo. « Polymer-Based Systems for Controlled Release and Targeting of Drugs ». Polymers 11, no 12 (11 décembre 2019) : 2066. http://dx.doi.org/10.3390/polym11122066.

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The current need to find new advanced approaches to carry biologically active substances (conventional organic drugs, peptides, proteins (such as antibodies), and nucleic acid-based drugs (NABDs such as siRNA and miRNA)) in the body fluids, to realize targeted therapies and even personalized ones, goes hand in hand with research on the performance of new materials to better realize appropriate drug vectors [...]
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Malik, Shipra, Brenda Asmara, Zoe Moscato, Jatinder Kaur Mukker et Raman Bahal. « Advances in Nanoparticle-based Delivery of Next Generation Peptide Nucleic Acids ». Current Pharmaceutical Design 24, no 43 (28 mars 2019) : 5164–74. http://dx.doi.org/10.2174/1381612825666190117164901.

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Background: Peptide nucleic acids (PNAs) belong to the next generation of synthetic nucleic acid analogues. Their high binding affinity and specificity towards the target DNA or RNA make them the reagent of choice for gene therapy-based applications. Objective: To review important gene therapy based applications of regular and chemically modified peptide nucleic acids in combination with nanotechnology. Method: Selective research of the literature. Results: Poor intracellular delivery of PNAs has been a significant challenge. Among several delivery strategies explored till date, nanotechnology-based strategies hold immense potential. Recent studies have shown that advances in nanotechnology can be used to broaden the range of therapeutic applications of PNAs. In this review, we discussed significant advances made in nanoparticle-based on PLGA polymer, silicon, oxidized carbon and graphene oxide for the delivery of PNAs. Conclusion: Nanoparticles delivered PNAs can be implied in diverse gene therapy based applications including gene editing as well as gene targeting (antisense) based strategies.
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Wang, Dong-Xia, Jing Wang, Ya-Xin Wang, Yi-Chen Du, Yan Huang, An-Na Tang, Yun-Xi Cui et De-Ming Kong. « DNA nanostructure-based nucleic acid probes : construction and biological applications ». Chemical Science 12, no 22 (2021) : 7602–22. http://dx.doi.org/10.1039/d1sc00587a.

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In recent years, DNA has been widely noted as a kind of material that can be used to construct building blocks for biosensing, in vivo imaging, drug development, and disease therapy because of its advantages of good biocompatibility and programmable properties.
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Chiarappa, Gianluca, Michela Abrami, Barbara Dapas, Rossella Farra, Fabio Trebez, Francesco Musiani, Gabriele Grassi et Mario Grassi. « Mathematical Modeling of Drug Release from Natural Polysaccharides Based Matrices ». Natural Product Communications 12, no 6 (juin 2017) : 1934578X1701200. http://dx.doi.org/10.1177/1934578x1701200610.

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The new concept of personalized medicine and the affirmation of Nucleic Acid Based Drugs (NABDs), an emerging class of bio-drugs constituted by short sequences of either DNA or RNA, represent a new challenge for the mathematical modelling in the drug delivery and adsorption field. Indeed, whether patient uniqueness asks for the use of theoretical tools enabling a rational approach adapting to each patient, NABDs delivery brings to our attention new aspects of drug delivery due to the NABDs fragile nature and way of action. This review aims to present and discuss the mathematical modelling of drug release from natural polysaccharides matrices with particular care to the description of the chemical and physical phenomena ruling drug delivery.
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Christopher, M., et J. Wong. « Recent Developments in Delivery of Nucleic Acid-Based Antiviral Agents ». Current Pharmaceutical Design 12, no 16 (1 juin 2006) : 1995–2006. http://dx.doi.org/10.2174/138161206777442146.

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Mishra, Sumita, Soyoun Kim et Dong-ki Lee. « Recent Patents on Nucleic Acid-Based Antiviral Therapeutics ». Recent Patents on Anti-Infective Drug Discovery 5, no 3 (1 novembre 2010) : 255–71. http://dx.doi.org/10.2174/157489110793348767.

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Seitz, Oliver. « Nucleic acid modification for fluorescence-based technologies ». Bioorganic & ; Medicinal Chemistry 16, no 1 (janvier 2008) : 17–18. http://dx.doi.org/10.1016/j.bmc.2007.04.061.

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Wang, Fei, Pan Li, Hoi Ching Chu et Pik Kwan Lo. « Nucleic Acids and Their Analogues for Biomedical Applications ». Biosensors 12, no 2 (4 février 2022) : 93. http://dx.doi.org/10.3390/bios12020093.

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Nucleic acids are emerging as powerful and functional biomaterials due to their molecular recognition ability, programmability, and ease of synthesis and chemical modification. Various types of nucleic acids have been used as gene regulation tools or therapeutic agents for the treatment of human diseases with genetic disorders. Nucleic acids can also be used to develop sensing platforms for detecting ions, small molecules, proteins, and cells. Their performance can be improved through integration with other organic or inorganic nanomaterials. To further enhance their biological properties, various chemically modified nucleic acid analogues can be generated by modifying their phosphodiester backbone, sugar moiety, nucleobase, or combined sites. Alternatively, using nucleic acids as building blocks for self-assembly of highly ordered nanostructures would enhance their biological stability and cellular uptake efficiency. In this review, we will focus on the development and biomedical applications of structural and functional natural nucleic acids, as well as the chemically modified nucleic acid analogues over the past ten years. The recent progress in the development of functional nanomaterials based on self-assembled DNA-based platforms for gene regulation, biosensing, drug delivery, and therapy will also be presented. We will then summarize with a discussion on the advanced development of nucleic acid research, highlight some of the challenges faced and propose suggestions for further improvement.
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Wiraja, Christian, David C. Yeo et Chenjie Xu. « Framework Nucleic Acids : A Paradigm Shift in Transdermal Drug Delivery ». SLAS TECHNOLOGY : Translating Life Sciences Innovation 24, no 5 (23 mai 2019) : 531–32. http://dx.doi.org/10.1177/2472630319848679.

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Transdermal drug delivery (TDD) provides a direct drug administration route bypassing gastrointestinal and liver metabolism. Until now, topical nanocarriers responsible for efficient TDD are predominantly polymeric or lipid based. The size-dependent skin penetration ability of framework nucleic acids (FNAs) has recently been reported, along with their efficacy in delivering doxorubicin for skin melanoma therapy. This commentary is to highlight the paradigm shift of nucleic acid delivery from being a cargo moiety to serving as a drug carrier instead. Further development directions to maximize the potential of FNAs for TDD are also discussed.
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Koyama, Shohei, Cevayir Coban, Taiki Aoshi, Toshihiro Horii, Shizuo Akira et Ken J. Ishii. « Innate immune control of nucleic acid-based vaccine immunogenicity ». Expert Review of Vaccines 8, no 8 (août 2009) : 1099–107. http://dx.doi.org/10.1586/erv.09.57.

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Labuda, Jan, Ana Maria Oliveira Brett, Gennady Evtugyn, Miroslav Fojta, Marco Mascini, Mehmet Ozsoz, Ilaria Palchetti, Emil Paleček et Joseph Wang. « Electrochemical nucleic acid-based biosensors : Concepts, terms, and methodology (IUPAC Technical Report) ». Pure and Applied Chemistry 82, no 5 (20 avril 2010) : 1161–87. http://dx.doi.org/10.1351/pac-rep-09-08-16.

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An electrochemical nucleic acid (NA)-based biosensor is a biosensor that integrates a nucleic acid as the biological recognition element and an electrode as the electrochemical signal transducer. The present report provides concepts, terms, and methodology related to biorecognition elements, detection principles, type of interactions to be addressed, and construction and performance of electrochemical NA biosensors, including their critical evaluation, which should be valuable for a wide audience, from academic, biomedical, environmental, and food-testing, drug-developing, etc. laboratories to sensor producers.
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Hueso, Miguel, Adrián Mallén, Marc Suñé-Pou, Josep M. Aran, Josep M. Suñé-Negre et Estanislao Navarro. « ncRNAs in Therapeutics : Challenges and Limitations in Nucleic Acid-Based Drug Delivery ». International Journal of Molecular Sciences 22, no 21 (27 octobre 2021) : 11596. http://dx.doi.org/10.3390/ijms222111596.

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Non-coding RNAs (ncRNAs) are emerging therapeutic tools but there are barriers to their translation to clinical practice. Key issues concern the specificity of the targets, the delivery of the molecules, and their stability, while avoiding “on-target” and “off-target” side effects. In this “ncRNA in therapeutics” issue, we collect several studies of the differential expression of ncRNAs in cardiovascular diseases, bone metabolism-related disorders, neurology, and oncology, and their potential to be used as biomarkers or therapeutic targets. Moreover, we review recent advances in the use of antisense ncRNAs in targeted therapies with a particular emphasis on their basic biological mechanisms, their translational potential, and future trends.
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Larkin, Joseph, Spencer Carson, Daniel H. Stoloff et Meni Wanunu. « Nanopore-Based Analysis of Chemically Modified DNA and Nucleic Acid Drug Targets ». Israel Journal of Chemistry 53, no 6-7 (juin 2013) : 431–41. http://dx.doi.org/10.1002/ijch.201300006.

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Wang, Jin, Tian Tian, Xin Li et Yan Zhang. « Noncoding RNAs Emerging as Drugs or Drug Targets : Their Chemical Modification, Bio-Conjugation and Intracellular Regulation ». Molecules 27, no 19 (9 octobre 2022) : 6717. http://dx.doi.org/10.3390/molecules27196717.

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With the increasing understanding of various disease-related noncoding RNAs, ncRNAs are emerging as novel drugs and drug targets. Nucleic acid drugs based on different types of noncoding RNAs have been designed and tested. Chemical modification has been applied to noncoding RNAs such as siRNA or miRNA to increase the resistance to degradation with minimum influence on their biological function. Chemical biological methods have also been developed to regulate relevant noncoding RNAs in the occurrence of various diseases. New strategies such as designing ribonuclease targeting chimeras to degrade endogenous noncoding RNAs are emerging as promising approaches to regulate gene expressions, serving as next-generation drugs. This review summarized the current state of noncoding RNA-based theranostics, major chemical modifications of noncoding RNAs to develop nucleic acid drugs, conjugation of RNA with different functional biomolecules as well as design and screening of potential molecules to regulate the expression or activity of endogenous noncoding RNAs for drug development. Finally, strategies of improving the delivery of noncoding RNAs are discussed.
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Valsangkar, Vibhav A., Arun Richard Chandrasekaran, Lifeng Zhuo, Song Mao, Goh Woon Lee, Megan Kizer, Xing Wang, Ken Halvorsen et Jia Sheng. « Click and photo-release dual-functional nucleic acid nanostructures ». Chemical Communications 55, no 65 (2019) : 9709–12. http://dx.doi.org/10.1039/c9cc03806j.

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Beg, Sarwar, Waleed H. Almalki, Fahmida Khatoon, Khalid S. Alharbi, Saad Alghamdi, Md Habban Akhter, Habibullah Khalilullah et al. « Lipid/polymer-based nanocomplexes in nucleic acid delivery as cancer vaccines ». Drug Discovery Today 26, no 8 (août 2021) : 1891–903. http://dx.doi.org/10.1016/j.drudis.2021.02.013.

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Ewert, Kai K., Pablo Scodeller, Lorena Simón-Gracia, Victoria M. Steffes, Emily A. Wonder, Tambet Teesalu et Cyrus R. Safinya. « Cationic Liposomes as Vectors for Nucleic Acid and Hydrophobic Drug Therapeutics ». Pharmaceutics 13, no 9 (30 août 2021) : 1365. http://dx.doi.org/10.3390/pharmaceutics13091365.

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Cationic liposomes (CLs) are effective carriers of a variety of therapeutics. Their applications as vectors of nucleic acids (NAs), from long DNA and mRNA to short interfering RNA (siRNA), have been pursued for decades to realize the promise of gene therapy, with approvals of the siRNA therapeutic patisiran and two mRNA vaccines against COVID-19 as recent milestones. The long-term goal of developing optimized CL-based NA carriers for a broad range of medical applications requires a comprehensive understanding of the structure of these vectors and their interactions with cell membranes and components that lead to the release and activity of the NAs within the cell. Structure–activity relationships of lipids for CL-based NA and drug delivery must take into account that these lipids act not individually but as components of an assembly of many molecules. This review summarizes our current understanding of how the choice of the constituting lipids governs the structure of their CL–NA self-assemblies, which constitute distinct liquid crystalline phases, and the relation of these structures to their efficacy for delivery. In addition, we review progress toward CL–NA nanoparticles for targeted NA delivery in vivo and close with an outlook on CL-based carriers of hydrophobic drugs, which may eventually lead to combination therapies with NAs and drugs for cancer and other diseases.
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Chen, Jing, Yue Tang, Yun Liu et Yushun Dou. « Nucleic Acid-Based Therapeutics for Pulmonary Diseases ». AAPS PharmSciTech 19, no 8 (18 octobre 2018) : 3670–80. http://dx.doi.org/10.1208/s12249-018-1183-0.

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Subjakova, Veronika, Veronika Oravczova et Tibor Hianik. « Polymer Nanoparticles and Nanomotors Modified by DNA/RNA Aptamers and Antibodies in Targeted Therapy of Cancer ». Polymers 13, no 3 (21 janvier 2021) : 341. http://dx.doi.org/10.3390/polym13030341.

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Polymer nanoparticles and nano/micromotors are novel nanostructures that are of increased interest especially in the diagnosis and therapy of cancer. These structures are modified by antibodies or nucleic acid aptamers and can recognize the cancer markers at the membrane of the cancer cells or in the intracellular side. They can serve as a cargo for targeted transport of drugs or nucleic acids in chemo- immuno- or gene therapy. The various mechanisms, such as enzyme, ultrasound, magnetic, electrical, or light, served as a driving force for nano/micromotors, allowing their transport into the cells. This review is focused on the recent achievements in the development of polymer nanoparticles and nano/micromotors modified by antibodies and nucleic acid aptamers. The methods of preparation of polymer nanoparticles, their structure and properties are provided together with those for synthesis and the application of nano/micromotors. The various mechanisms of the driving of nano/micromotors such as chemical, light, ultrasound, electric and magnetic fields are explained. The targeting drug delivery is based on the modification of nanostructures by receptors such as nucleic acid aptamers and antibodies. Special focus is therefore on the method of selection aptamers for recognition cancer markers as well as on the comparison of the properties of nucleic acid aptamers and antibodies. The methods of immobilization of aptamers at the nanoparticles and nano/micromotors are provided. Examples of applications of polymer nanoparticles and nano/micromotors in targeted delivery and in controlled drug release are presented. The future perspectives of biomimetic nanostructures in personalized nanomedicine are also discussed.
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Kim, Hyosuk, Hochung Jang, Haeun Cho, Jiwon Choi, Kwang Yeon Hwang, Yeonho Choi, Sun Hwa Kim et Yoosoo Yang. « Recent Advances in Exosome-Based Drug Delivery for Cancer Therapy ». Cancers 13, no 17 (2 septembre 2021) : 4435. http://dx.doi.org/10.3390/cancers13174435.

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Exosomes are a class of extracellular vesicles, with a size of about 100 nm, secreted by most cells and carrying various bioactive molecules such as nucleic acids, proteins, and lipids, and reflect the biological status of parent cells. Exosomes have natural advantages such as high biocompatibility and low immunogenicity for efficient delivery of therapeutic agents such as chemotherapeutic drugs, nucleic acids, and proteins. In this review, we introduce the latest explorations of exosome-based drug delivery systems for cancer therapy, with particular focus on the targeted delivery of various types of cargoes.
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Dai, Ziwen, Juan Li, Yongfang Lin, Zhigang Wang et Yang Huang. « Facile Construction of a Solely-DNA-Based System for Targeted Delivery of Nucleic Acids ». Nanomaterials 11, no 8 (30 juillet 2021) : 1967. http://dx.doi.org/10.3390/nano11081967.

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We designed a functional drug delivery system based solely on DNA. The whole system was built with only four DNA strands. Cyclization of DNA strands excluded the formation of byproducts. DNA aptamers were equipped to endow triangular DNA nanostructures with targeting ability. The homogeneity of materials enabled not only facile construction but also convenient loading of nucleic acid-based drugs with much ease.
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Zhang, Chi, Yifan Ma, Jingjing Zhang, Jimmy Chun-Tien Kuo, Zhongkun Zhang, Haotian Xie, Jing Zhu et Tongzheng Liu. « Modification of Lipid-Based Nanoparticles : An Efficient Delivery System for Nucleic Acid-Based Immunotherapy ». Molecules 27, no 6 (17 mars 2022) : 1943. http://dx.doi.org/10.3390/molecules27061943.

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Lipid-based nanoparticles (LBNPs) are biocompatible and biodegradable vesicles that are considered to be one of the most efficient drug delivery platforms. Due to the prominent advantages, such as long circulation time, slow drug release, reduced toxicity, high transfection efficiency, and endosomal escape capacity, such synthetic nanoparticles have been widely used for carrying genetic therapeutics, particularly nucleic acids that can be applied in the treatment for various diseases, including congenital diseases, cancers, virus infections, and chronic inflammations. Despite great merits and multiple successful applications, many extracellular and intracellular barriers remain and greatly impair delivery efficacy and therapeutic outcomes. As such, the current state of knowledge and pitfalls regarding the gene delivery and construction of LBNPs will be initially summarized. In order to develop a new generation of LBNPs for improved delivery profiles and therapeutic effects, the modification strategies of LBNPs will be reviewed. On the basis of these developed modifications, the performance of LBNPs as therapeutic nanoplatforms have been greatly improved and extensively applied in immunotherapies, including infectious diseases and cancers. However, the therapeutic applications of LBNPs systems are still limited due to the undesirable endosomal escape, potential aggregation, and the inefficient encapsulation of therapeutics. Herein, we will review and discuss recent advances and remaining challenges in the development of LBNPs for nucleic acid-based immunotherapy.
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Aziz Ahmad, Kashif, Saleha Akram Nizami et Muhammad Haroon Ghous. « Coronavirus - Drug Discovery and Therapeutic Drug Monitoring Options ». Pharmaceutics and Pharmacology Research 5, no 2 (6 janvier 2022) : 01–04. http://dx.doi.org/10.31579/2693-7247/044.

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COVID-19 is basically a medium size RNA virus and the nucleic acid is about 30 kb long, positive in sense, single stranded and polyadenylated. The RNA which is found in this virus is the largest known RNA and codes for a large polyprotein. In addition, coronaviruses are capable of genetic recombination if 2 viruses infect the same cell at the same time. SARS-CoV emerged first in southern China and rapidly spread around the globe in 2002–2003. In November 2002, an unusual epidemic of atypical pneumonia with a high rate of nosocomial transmission to health-care workers occurred in Foshan, Guangdong, China. In March 2003, a novel CoV was confirmed to be the causative agent for SARS, and was thus named SARS-CoV. Despite the report of a large number of virus-based and host-based treatment options with potent in vitro activities for SARS and MERS, only a few are likely to fulfil their potential in the clinical setting in the foreseeable future. Most drugs have one or more major limitations that prevent them from proceeding beyond the in vitro stage. First, many drugs have high EC50/Cmax ratios at clinically relevant dosages
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