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Thematische Bibliographien / GENE DELIVERY APPLICATIONS / Zeitschriftenartikel

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Zeitschriftenartikel zum Thema „GENE DELIVERY APPLICATIONS“

Autor: Grafiati

Veröffentlicht am 11. September 2023

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1

Huang, Rih-Yang, Zhuo-Hao Liu, Wei-Han Weng und Chien-Wen Chang. „Magnetic nanocomplexes for gene delivery applications“. Journal of Materials Chemistry B 9, Nr. 21 (2021): 4267–86. http://dx.doi.org/10.1039/d0tb02713h.

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This review paper covers the recent progress of magnetic nanoparticles (MNP)-based gene delivery. Cutting-edge applications of MNP-based gene delivery on cancer therapy, neural repairing, regenerative medicine and gene editing are also introduced.

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Chen, Chih-Kuang, Ping-Kuan Huang, Wing-Cheung Law, Chia-Hui Chu, Nai-Tzu Chen und Leu-Wei Lo. „Biodegradable Polymers for Gene-Delivery Applications“. International Journal of Nanomedicine Volume 15 (März 2020): 2131–50. http://dx.doi.org/10.2147/ijn.s222419.

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3

Katz, M. G., A. S. Fargnoli, L. A. Pritchette und C. R. Bridges. „Gene delivery technologies for cardiac applications“. Gene Therapy 19, Nr. 6 (15.03.2012): 659–69. http://dx.doi.org/10.1038/gt.2012.11.

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4

Makkonen, Kaisa-Emilia, Kari Airenne und Seppo Ylä-Herttulala. „Baculovirus-mediated Gene Delivery and RNAi Applications“. Viruses 7, Nr. 4 (22.04.2015): 2099–125. http://dx.doi.org/10.3390/v7042099.

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5

Suda, Takeshi, und Dexi Liu. „Hydrodynamic Gene Delivery: Its Principles and Applications“. Molecular Therapy 15, Nr. 12 (Dezember 2007): 2063–69. http://dx.doi.org/10.1038/sj.mt.6300314.

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6

Yin, Feng, Bobo Gu, Yining Lin, Nishtha Panwar, Swee Chuan Tjin, Junle Qu, Shu Ping Lau und Ken-Tye Yong. „Functionalized 2D nanomaterials for gene delivery applications“. Coordination Chemistry Reviews 347 (September 2017): 77–97. http://dx.doi.org/10.1016/j.ccr.2017.06.024.

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7

Rabiee, Navid, Shokooh Ahmadvand, Sepideh Ahmadi, Yousef Fatahi, Rassoul Dinarvand, Mojtaba Bagherzadeh, Mohammad Rabiee, Mohammadreza Tahriri, Lobat Tayebi und Michael R. Hamblin. „Carbosilane dendrimers: Drug and gene delivery applications“. Journal of Drug Delivery Science and Technology 59 (Oktober 2020): 101879. http://dx.doi.org/10.1016/j.jddst.2020.101879.

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8

Wich, Peter R., und Jean M. J. Fréchet. „Degradable Dextran Particles for Gene Delivery Applications“. Australian Journal of Chemistry 65, Nr. 1 (2012): 15. http://dx.doi.org/10.1071/ch11370.

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Successful gene therapy depends both on the effective transport and the stable expression of therapeutic genes to produce and regulate disease related proteins. In this context, non-viral gene delivery vehicles are regarded as one of the most promising approaches for the efficient and safe transport of genetic material to and into the target cells. This short review describes the development of novel particulate delivery vehicles based on the biopolymer dextran. This multifunctional platform was designed to safely transport genetic material across cell membranes, followed by an acid triggered release that causes overall high transfection efficiency. The biocompatibility and its unique tunability differentiate this new carrier system from previous particle systems, showing high potential for the treatment of several disease models in RNA interference related applications.

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Kafshdooz, Taiebeh, Leila Kafshdooz, Abolfazl Akbarzadeh, Younes Hanifehpour und Sang Woo Joo. „Applications of nanoparticle systems in gene delivery and gene therapy“. Artificial Cells, Nanomedicine, and Biotechnology 44, Nr. 2 (03.11.2014): 581–87. http://dx.doi.org/10.3109/21691401.2014.971805.

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10

Contin, Mario, Cybele Garcia, Cecilia Dobrecky, Silvia Lucangioli und Norma D’Accorso. „Advances in drug delivery, gene delivery and therapeutic agents based on dendritic materials“. Future Medicinal Chemistry 11, Nr. 14 (Juli 2019): 1791–810. http://dx.doi.org/10.4155/fmc-2018-0452.

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Dendrimers are synthetic polymers that grow in three dimensions into well-defined structures. Their morphological appearance resembles a number of trees connected by a common point. Dendritic nanoparticles have been studied for a large number of pharmaceutical and biomedical applications including gene and drug delivery, clinical diagnosis and MRI. Despite the application of dendrimers, research is still in its childhood in comparison with liposomes and other nanomaterials. They are now playing a key role in several therapeutic strategies, with dendrimer-based products in clinical trials. The aim of this review is to describe the state-of-the-art of biomedical applications of dendrimers – and dendrimer conjugates – such as drug and gene delivery and antiviral activity.

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Zhang, Chao, Shanhe Liu, Xuan Li, Ruixuan Zhang und Jun Li. „Virus-Induced Gene Editing and Its Applications in Plants“. International Journal of Molecular Sciences 23, Nr. 18 (06.09.2022): 10202. http://dx.doi.org/10.3390/ijms231810202.

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CRISPR/Cas-based genome editing technologies, which allow the precise manipulation of plant genomes, have revolutionized plant science and enabled the creation of germplasms with beneficial traits. In order to apply these technologies, CRISPR/Cas reagents must be delivered into plant cells; however, this is limited by tissue culture challenges. Recently, viral vectors have been used to deliver CRISPR/Cas reagents into plant cells. Virus-induced genome editing (VIGE) has emerged as a powerful method with several advantages, including high editing efficiency and a simplified process for generating gene-edited DNA-free plants. Here, we briefly describe CRISPR/Cas-based genome editing. We then focus on VIGE systems and the types of viruses used currently for CRISPR/Cas9 cassette delivery and genome editing. We also highlight recent applications of and advances in VIGE in plants. Finally, we discuss the challenges and potential for VIGE in plants.

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Mantz, Amy, und Angela K. Pannier. „Biomaterial substrate modifications that influence cell-material interactions to prime cellular responses to nonviral gene delivery“. Experimental Biology and Medicine 244, Nr. 2 (08.01.2019): 100–113. http://dx.doi.org/10.1177/1535370218821060.

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Gene delivery is the transfer of exogenous genetic material into somatic cells to modify their gene expression, with applications including tissue engineering, regenerative medicine, sensors and diagnostics, and gene therapy. Viral vectors are considered the most effective system to deliver nucleic acids, yet safety concerns and many other disadvantages have resulted in investigations into an alternative option, i.e. nonviral gene delivery. Chemical nonviral gene delivery is typically accomplished by electrostatically complexing cationic lipids or polymers with negatively charged nucleic acids. Unfortunately, nonviral gene delivery suffers from low efficiency due to barriers that impede transfection success, including intracellular processes such as internalization, endosomal escape, cytosolic trafficking, and nuclear entry. Efforts to improve nonviral gene delivery have focused on modifying nonviral vectors, yet a novel solution that may prove more effective than vector modifications is stimulating or “priming” cells before transfection to modulate and mitigate the cellular response to nonviral gene delivery. In applications where a cell-material interface exists, cell priming can come from cues from the substrate, through chemical modifications such as the addition of natural coatings, ligands, or functional side groups, and/or physical modifications such as topography or stiffness, to mimic extracellular matrix cues and modulate cellular behaviors that influence transfection efficiency. This review summarizes how biomaterial substrate modifications can prime the cellular response to nonviral gene delivery (e.g. integrin binding and focal adhesion formation, cytoskeletal remodeling, endocytic mechanisms, intracellular trafficking) to aid in improving gene delivery for future therapeutic applications. Impact statement This review summarizes how biomaterial substrate modifications (e.g. chemical modifications like natural coatings, ligands, or functional side groups, and/or physical modifications such as topography or stiffness) can prime the cellular response to nonviral gene delivery (e.g. affecting integrin binding and focal adhesion formation, cytoskeletal remodeling, endocytic mechanisms, and intracellular trafficking), to aid in improving gene delivery for applications where a cell-material interface might exist (e.g. tissue engineering scaffolds, medical implants and devices, sensors and diagnostics, wound dressings).

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Zhou, Qiu-Lan, Zhi-Yi Chen, Yi-Xiang Wang, Feng Yang, Yan Lin und Yang-Ying Liao. „Ultrasound-Mediated Local Drug and Gene Delivery Using Nanocarriers“. BioMed Research International 2014 (2014): 1–13. http://dx.doi.org/10.1155/2014/963891.

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With the development of nanotechnology, nanocarriers have been increasingly used for curative drug/gene delivery. Various nanocarriers are being introduced and assessed, such as polymer nanoparticles, liposomes, and micelles. As a novel theranostic system, nanocarriers hold great promise for ultrasound molecular imaging, targeted drug/gene delivery, and therapy. Nanocarriers, with the properties of smaller particle size, and long circulation time, would be advantageous in diagnostic and therapeutic applications. Nanocarriers can pass through blood capillary walls and cell membrane walls to deliver drugs. The mechanisms of interaction between ultrasound and nanocarriers are not clearly understood, which may be related to cavitation, mechanical effects, thermal effects, and so forth. These effects may induce transient membrane permeabilization (sonoporation) on a single cell level, cell death, and disruption of tissue structure, ensuring noninvasive, targeted, and efficient drug/gene delivery and therapy. The system has been used in various tissues and organs (in vitro or in vivo), including tumor tissues, kidney, cardiac, skeletal muscle, and vascular smooth muscle. In this review, we explore the research progress and application of ultrasound-mediated local drug/gene delivery with nanocarriers.

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Pise, Ajay G. „Archaeosomes for both cell-based delivery applications and drug-based delivery applications“. Journal of medical pharmaceutical and allied sciences 11, Nr. 3 (30.06.2022): 4995–5003. http://dx.doi.org/10.55522/jmpas.v11i3.2471.

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Archaeosomes, or liposomes produced with one or more Archaeobacteria-specific ether lipids, are a novel kind of liposome found in Archaea. The fundamental structures of Achaean-type lipids are archaeol (diether) and/or caldarchaeol (tetraether). To make archaeosomes at any temperature in the physiological range or lower which enable thermally stable compounds to be encapsulated, traditional procedures like hydrated film sonicated, extrusion, and detergent dialysis are used. A multitude of physiological and environmental factors impact its stability. Archaeosomes are widely used as drug delivery systems for cancer vaccines, Chagas disease, proteins and peptides, gene delivery, antigen delivery, and administration of natural antioxidant compounds. The major purpose of this study was to look at how this unique carrier technology may be used in the pharmaceutical industry.

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Kumar, Raj, Arun Butreddy, Nagavendra Kommineni, Pulikanti Guruprasad Reddy, Naveen Bunekar, Chandrani Sarkar, Sunil Dutt et al. „Lignin: Drug/Gene Delivery and Tissue Engineering Applications“. International Journal of Nanomedicine Volume 16 (März 2021): 2419–41. http://dx.doi.org/10.2147/ijn.s303462.

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16

Keles, Erhan, Yang Song, Dan Du, Wen-Ji Dong und Yuehe Lin. „Recent progress in nanomaterials for gene delivery applications“. Biomaterials Science 4, Nr. 9 (2016): 1291–309. http://dx.doi.org/10.1039/c6bm00441e.

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17

Ojea-Jiménez, Isaac, Olivia Tort, Julia Lorenzo und Victor F. Puntes. „Engineered nonviral nanocarriers for intracellular gene delivery applications“. Biomedical Materials 7, Nr. 5 (12.09.2012): 054106. http://dx.doi.org/10.1088/1748-6041/7/5/054106.

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18

Cifuentes-Rius, Anna, Ana de Pablo, Victor Ramos-Pérez und Salvador Borrós. „Tailoring Carbon Nanotubes Surface for Gene Delivery Applications“. Plasma Processes and Polymers 11, Nr. 7 (16.06.2014): 704–13. http://dx.doi.org/10.1002/ppap.201300167.

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19

Xiu, Kemao, Jifeng Zhang, Jie Xu, Y. Eugene Chen und Peter X. Ma. „Recent progress in polymeric gene vectors: Delivery mechanisms, molecular designs, and applications“. Biophysics Reviews 4, Nr. 1 (März 2023): 011313. http://dx.doi.org/10.1063/5.0123664.

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Gene therapy and gene delivery have drawn extensive attention in recent years especially when the COVID-19 mRNA vaccines were developed to prevent severe symptoms caused by the corona virus. Delivering genes, such as DNA and RNA into cells, is the crucial step for successful gene therapy and remains a bottleneck. To address this issue, vehicles (vectors) that can load and deliver genes into cells are developed, including viral and non-viral vectors. Although viral gene vectors have considerable transfection efficiency and lipid-based gene vectors become popular since the application of COVID-19 vaccines, their potential issues including immunologic and biological safety concerns limited their applications. Alternatively, polymeric gene vectors are safer, cheaper, and more versatile compared to viral and lipid-based vectors. In recent years, various polymeric gene vectors with well-designed molecules were developed, achieving either high transfection efficiency or showing advantages in certain applications. In this review, we summarize the recent progress in polymeric gene vectors including the transfection mechanisms, molecular designs, and biomedical applications. Commercially available polymeric gene vectors/reagents are also introduced. Researchers in this field have never stopped seeking safe and efficient polymeric gene vectors via rational molecular designs and biomedical evaluations. The achievements in recent years have significantly accelerated the progress of polymeric gene vectors toward clinical applications.

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JAIN, SHARDOOL, HUSAIN ATTARWALA und MANSOOR AMIJI. „NON-CONDENSING POLYMERIC GENE DELIVERY SYSTEMS: PRINCIPLES AND APPLICATIONS“. Nano LIFE 01, Nr. 03n04 (September 2010): 219–37. http://dx.doi.org/10.1142/s1793984410000249.

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Gene therapy holds tremendous promise in prevention and treatment of diseases as the approach is based on regulating the expression of genes that are responsible for pathological conditions. The biggest bottleneck for gene delivery has been the development of safe and efficacious delivery systems. Although non-viral vectors are considered as much safer options than their viral counterparts, they suffer from low transfection efficiency. In this review, we highlight the role of non-condensing polymeric delivery systems for oral and systemic gene delivery. Using evidence from contemporary literature, non-condensing polymeric microparticle and nanoparticle systems afford physical encapsulation of the nucleic acid construct and can be engineered for targeted delivery to tissues and cells. Additionally, these systems have shown less toxicity and afford sustained cytoplasmic DNA delivery for efficient nuclear uptake and transfection for both DNA vaccines and therapeutic genes.

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Chen, Qiang, und Huafang Lai. „Gene Delivery into Plant Cells for Recombinant Protein Production“. BioMed Research International 2015 (2015): 1–10. http://dx.doi.org/10.1155/2015/932161.

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Recombinant proteins are primarily produced from cultures of mammalian, insect, and bacteria cells. In recent years, the development of deconstructed virus-based vectors has allowed plants to become a viable platform for recombinant protein production, with advantages in versatility, speed, cost, scalability, and safety over the current production paradigms. In this paper, we review the recent progress in the methodology of agroinfiltration, a solution to overcome the challenge of transgene delivery into plant cells for large-scale manufacturing of recombinant proteins. General gene delivery methodologies in plants are first summarized, followed by extensive discussion on the application and scalability of each agroinfiltration method. New development of a spray-based agroinfiltration and its application on field-grown plants is highlighted. The discussion of agroinfiltration vectors focuses on their applications for producing complex and heteromultimeric proteins and is updated with the development of bridge vectors. Progress on agroinfiltration inNicotianaand non-Nicotianaplant hosts is subsequently showcased in context of their applications for producing high-value human biologics and low-cost and high-volume industrial enzymes. These new advancements in agroinfiltration greatly enhance the robustness and scalability of transgene delivery in plants, facilitating the adoption of plant transient expression systems for manufacturing recombinant proteins with a broad range of applications.

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Malik, Shipra, Brenda Asmara, Zoe Moscato, Jatinder Kaur Mukker und Raman Bahal. „Advances in Nanoparticle-based Delivery of Next Generation Peptide Nucleic Acids“. Current Pharmaceutical Design 24, Nr. 43 (28.03.2019): 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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Wu, Jiangyu, Weizhe Huang und Ziying He. „Dendrimers as Carriers for siRNA Delivery and Gene Silencing: A Review“. Scientific World Journal 2013 (2013): 1–16. http://dx.doi.org/10.1155/2013/630654.

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RNA interference (RNAi) was first literaturally reported in 1998 and has become rapidly a promising tool for therapeutic applications in gene therapy. In a typical RNAi process, small interfering RNAs (siRNA) are used to specifically downregulate the expression of the targeted gene, known as the term “gene silencing.” One key point for successful gene silencing is to employ a safe and efficient siRNA delivery system. In this context, dendrimers are emerging as potential nonviral vectors to deliver siRNA for RNAi purpose. Dendrimers have attracted intense interest since their emanating research in the 1980s and are extensively studied as efficient DNA delivery vectors in gene transfer applications, due to their unique features based on the well-defined and multivalent structures. Knowing that DNA and RNA possess a similar structure in terms of nucleic acid framework and the electronegative nature, one can also use the excellent DNA delivery properties of dendrimers to develop effective siRNA delivery systems. In this review, the development of dendrimer-based siRNA delivery vectors is summarized, focusing on the vector features (siRNA delivery efficiency, cytotoxicity, etc.) of different types of dendrimers and the related investigations on structure-activity relationship to promote safe and efficient siRNA delivery system.

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Warga, Eric, Brian Austin-Carter, Noelle Comolli und Jacob Elmer. „Nonviral Vehicles for Gene Delivery“. Nano LIFE 11, Nr. 02 (Juni 2021): 2130002. http://dx.doi.org/10.1142/s1793984421300028.

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Nonviral gene delivery (NVGD) is an appealing alternative to viral gene delivery for clinical applications due to its lower cost and increased safety. A variety of promising nonviral vectors are under development, including cationic polymers, lipids, lipid-polymer hybrids (LPHs) and inorganic nanoparticles. However, some NVGD strategies have disadvantages that have limited their adoption, including high toxicity and low efficiency. This review focuses on the most common NVGD vehicles with an emphasis on recent developments in the field.

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Wu, Huiming. „Current Development of the Applications of Polymers in Gene Delivery“. E3S Web of Conferences 271 (2021): 04043. http://dx.doi.org/10.1051/e3sconf/202127104043.

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Gene delivery is one of the most important and efficient therapy methods for treating diseases, especially genetic diseases that challenge conventional treatment methods. However, to be efficient, gene delivery has a few requirements for the polymers to be applicable, including the reduction of cytotoxicity, the improvement of transfection efficiency, and the elimination of off-target effects. In this study, the common polymers applied in gene delivery is discussed in depth, regarding such requirements, as well as some of the limitations and potential improvements in gene delivery.

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Er, Simge, Ushna Laraib, Rabia Arshad, Saman Sargazi, Abbas Rahdar, Sadanand Pandey, Vijay Kumar Thakur und Ana M. Díez-Pascual. „Amino Acids, Peptides, and Proteins: Implications for Nanotechnological Applications in Biosensing and Drug/Gene Delivery“. Nanomaterials 11, Nr. 11 (08.11.2021): 3002. http://dx.doi.org/10.3390/nano11113002.

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Over various scientific fields in biochemistry, amino acids have been highlighted in research works. Protein, peptide- and amino acid-based drug delivery systems have proficiently transformed nanotechnology via immense flexibility in their features for attaching various drug molecules and biodegradable polymers. In this regard, novel nanostructures including carbon nanotubes, electrospun carbon nanofibers, gold nanoislands, and metal-based nanoparticles have been introduced as nanosensors for accurate detection of these organic compounds. These nanostructures can bind the biological receptor to the sensor surface and increase the surface area of the working electrode, significantly enhancing the biosensor performance. Interestingly, protein-based nanocarriers have also emerged as useful drug and gene delivery platforms. This is important since, despite recent advancements, there are still biological barriers and other obstacles limiting gene and drug delivery efficacy. Currently available strategies for gene therapy are not cost-effective, and they do not deliver the genetic cargo effectively to target sites. With rapid advancements in nanotechnology, novel gene delivery systems are introduced as nonviral vectors such as protein, peptide, and amino acid-based nanostructures. These nano-based delivery platforms can be tailored into functional transformation using proteins and peptides ligands based nanocarriers, usually overexpressed in the specified diseases. The purpose of this review is to shed light on traditional and nanotechnology-based methods to detect amino acids, peptides, and proteins. Furthermore, new insights into the potential of amino protein-based nanoassemblies for targeted drug delivery or gene transfer are presented.

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Cui, Zhanpeng, Yang Jiao, Linyu Pu, James Zhenggui Tang und Gang Wang. „The Progress of Non-Viral Materials and Methods for Gene Delivery to Skeletal Muscle“. Pharmaceutics 14, Nr. 11 (10.11.2022): 2428. http://dx.doi.org/10.3390/pharmaceutics14112428.

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Since Jon A. Wolff found skeletal muscle cells being able to express foreign genes and Russell J. Mumper increased the gene transfection efficiency into the myocytes by adding polymers, skeletal muscles have become a potential gene delivery and expression target. Different methods have been developing to deliver transgene into skeletal muscles. Among them, viral vectors may achieve potent gene delivery efficiency. However, the potential for triggering biosafety risks limited their clinical applications. Therefore, non-viral biomaterial-mediated methods with reliable biocompatibility are promising tools for intramuscular gene delivery in situ. In recent years, a series of advanced non-viral gene delivery materials and related methods have been reported, such as polymers, liposomes, cell penetrating peptides, as well as physical delivery methods. In this review, we summarized the research progresses and challenges in non-viral intramuscular gene delivery materials and related methods, focusing on the achievements and future directions of polymers.

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Jahangiri-Manesh, Atieh, Marziyeh Mousazadeh, Shirinsadat Taji, Abbas Bahmani, Atefeh Zarepour, Ali Zarrabi, Esmaeel Sharifi und Mostafa Azimzadeh. „Gold Nanorods for Drug and Gene Delivery: An Overview of Recent Advancements“. Pharmaceutics 14, Nr. 3 (17.03.2022): 664. http://dx.doi.org/10.3390/pharmaceutics14030664.

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Over the past few decades, gold nanomaterials have shown great promise in the field of nanotechnology, especially in medical and biological applications. They have become the most used nanomaterials in those fields due to their several advantageous. However, rod-shaped gold nanoparticles, or gold nanorods (GNRs), have some more unique physical, optical, and chemical properties, making them proper candidates for biomedical applications including drug/gene delivery, photothermal/photodynamic therapy, and theranostics. Most of their therapeutic applications are based on their ability for tunable heat generation upon exposure to near-infrared (NIR) radiation, which is helpful in both NIR-responsive cargo delivery and photothermal/photodynamic therapies. In this review, a comprehensive insight into the properties, synthesis methods and toxicity of gold nanorods are overviewed first. For the main body of the review, the therapeutic applications of GNRs are provided in four main sections: (i) drug delivery, (ii) gene delivery, (iii) photothermal/photodynamic therapy, and (iv) theranostics applications. Finally, the challenges and future perspectives of their therapeutic application are discussed.

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Singh, Swita R., und Uday B. Kompella. „Nanotechnology for Gene Delivery to the Eye“. European Ophthalmic Review 03, Nr. 01 (2009): 7. http://dx.doi.org/10.17925/eor.2009.03.01.7.

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The relatively immune-privileged status of the eye makes it an interesting target for gene delivery. Gene delivery to the eye using viral vectors via subretinal and intravitreal injections has been extensively investigated. Recently, the safety of recombinant adeno-associated virus vector expressing RPE65 complementary DNA (cDNA) in a limited clinical trial of three patients has also been reported. Nanotechnology-based non-viral vectors offer the advantages of safety and flexibility in terms of loading capacity and delivery system design compared with viral vectors. An ideal non-viral vector should be non-toxic, efficiently taken up into the target cells and conducive to gene expression, and should protect the gene against enzymatic degradation. Multiple kinds of nanotechnology-based non-viral vectors have been investigated for potential applications for gene delivery to the eye, namely nanoplexes, dendrimers, micelles, nanoparticles and liposomes. This article summarises and discusses key advances in the application of nanotechnology for gene delivery to the eye.

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Bastola, Prabhakar, Liujiang Song, Brian C. Gilger und Matthew L. Hirsch. „Adeno-Associated Virus Mediated Gene Therapy for Corneal Diseases“. Pharmaceutics 12, Nr. 8 (13.08.2020): 767. http://dx.doi.org/10.3390/pharmaceutics12080767.

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According to the World Health Organization, corneal diseases are the fourth leading cause of blindness worldwide accounting for 5.1% of all ocular deficiencies. Current therapies for corneal diseases, which include eye drops, oral medications, corrective surgeries, and corneal transplantation are largely inadequate, have undesirable side effects including blindness, and can require life-long applications. Adeno-associated virus (AAV) mediated gene therapy is an optimistic strategy that involves the delivery of genetic material to target human diseases through gene augmentation, gene deletion, and/or gene editing. With two therapies already approved by the United States Food and Drug Administration and 200 ongoing clinical trials, recombinant AAV (rAAV) has emerged as the in vivo viral vector-of-choice to deliver genetic material to target human diseases. Likewise, the relative ease of applications through targeted delivery and its compartmental nature makes the cornea an enticing tissue for AAV mediated gene therapy applications. This current review seeks to summarize the development of AAV gene therapy, highlight preclinical efficacy studies, and discuss potential applications and challenges of this technology for targeting corneal diseases.

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Gutiérrez, Sergio, und Kyle J. Lauersen. „Gene Delivery Technologies with Applications in Microalgal Genetic Engineering“. Biology 10, Nr. 4 (26.03.2021): 265. http://dx.doi.org/10.3390/biology10040265.

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Microalgae and cyanobacteria are photosynthetic microbes that can be grown with the simple inputs of water, carbon dioxide, (sun)light, and trace elements. Their engineering holds the promise of tailored bio-molecule production using sustainable, environmentally friendly waste carbon inputs. Although algal engineering examples are beginning to show maturity, severe limitations remain in the transformation of multigene expression cassettes into model species and DNA delivery into non-model hosts. This review highlights common and emerging DNA delivery methods used for other organisms that may find future applications in algal engineering.

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Alhakamy, Nabil A., Adane S. Nigatu, Cory J. Berkland und Joshua D. Ramsey. „Noncovalently associated cell-penetrating peptides for gene delivery applications“. Therapeutic Delivery 4, Nr. 6 (Juni 2013): 741–57. http://dx.doi.org/10.4155/tde.13.44.

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Sitta, Juliana, und Candace M. Howard. „Applications of Ultrasound-Mediated Drug Delivery and Gene Therapy“. International Journal of Molecular Sciences 22, Nr. 21 (25.10.2021): 11491. http://dx.doi.org/10.3390/ijms222111491.

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Gene therapy has continuously evolved throughout the years since its first proposal to develop more specific and effective transfection, capable of treating a myriad of health conditions. Viral vectors are some of the most common and most efficient vehicles for gene transfer. However, the safe and effective delivery of gene therapy remains a major obstacle. Ultrasound contrast agents in the form of microbubbles have provided a unique solution to fulfill the need to shield the vectors from the host immune system and the need for site specific targeted therapy. Since the discovery of the biophysical and biological effects of microbubble sonification, multiple developments have been made to enhance its applicability in targeted drug delivery. The concurrent development of viral vectors and recent research on dual vector strategies have shown promising results. This review will explore the mechanisms and recent advancements in the knowledge of ultrasound-mediated microbubbles in targeting gene and drug therapy.

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Raftery, Rosanne, Fergal O'Brien und Sally-Ann Cryan. „Chitosan for Gene Delivery and Orthopedic Tissue Engineering Applications“. Molecules 18, Nr. 5 (15.05.2013): 5611–47. http://dx.doi.org/10.3390/molecules18055611.

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Imani, Rana, Fatemeh Mohabatpour und Fatemeh Mostafavi. „Graphene-based Nano-Carrier modifications for gene delivery applications“. Carbon 140 (Dezember 2018): 569–91. http://dx.doi.org/10.1016/j.carbon.2018.09.019.

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Litzinger, David C., und Leaf Huang. „Phosphatodylethanolamine liposomes: drug delivery, gene transfer and immunodiagnostic applications“. Biochimica et Biophysica Acta (BBA) - Reviews on Biomembranes 1113, Nr. 2 (August 1992): 201–27. http://dx.doi.org/10.1016/0304-4157(92)90039-d.

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Flick, E., A. Belski, Wenzhong Li, G. Steinhoff und H. H. Gatzen. „Magnetic Microactuator for Controlling Nanoparticles in Gene Delivery Applications“. IEEE Transactions on Magnetics 45, Nr. 10 (Oktober 2009): 4869–72. http://dx.doi.org/10.1109/tmag.2009.2025669.

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Lee, Jun-hyung, Young Joo Choi und Yong-beom Lim. „Self-assembled filamentous nanostructures for drug/gene delivery applications“. Expert Opinion on Drug Delivery 7, Nr. 3 (März 2010): 341–51. http://dx.doi.org/10.1517/17425240903559841.

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Paliwal, Sumit, und Samir Mitragotri. „Ultrasound-induced cavitation: applications in drug and gene delivery“. Expert Opinion on Drug Delivery 3, Nr. 6 (31.10.2006): 713–26. http://dx.doi.org/10.1517/17425247.3.6.713.

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Chen, Hao H., Pratiek N. Matkar, Kolsoom Afrasiabi, Michael A. Kuliszewski und Howard Leong-Poi. „Prospect of ultrasound-mediated gene delivery in cardiovascular applications“. Expert Opinion on Biological Therapy 16, Nr. 6 (11.04.2016): 815–26. http://dx.doi.org/10.1517/14712598.2016.1169268.

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Arote, Rohidas B., Dhananjay Jere, Hu-Lin Jiang, You-Kyoung Kim, Yun-Jaie Choi und Chong-Su Cho. „Biodegradable poly(ester amine)s for gene delivery applications“. Biomedical Materials 4, Nr. 4 (08.07.2009): 044102. http://dx.doi.org/10.1088/1748-6041/4/4/044102.

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Byk, Gerardo, Catherine Dubertret, Bertrand Schwartz, Marc Frederic, Gabrielle Jaslin, Ravi Rangara und Daniel Scherman. „Novel nonviral vectors for gene delivery: Synthesis and applications“. Letters in Peptide Science 4, Nr. 4-6 (Dezember 1997): 263–67. http://dx.doi.org/10.1007/bf02442887.

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Bonamassa, Barbara, Li Hai und Dexi Liu. „Hydrodynamic Gene Delivery and Its Applications in Pharmaceutical Research“. Pharmaceutical Research 28, Nr. 4 (30.12.2010): 694–701. http://dx.doi.org/10.1007/s11095-010-0338-9.

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Chuan, Di, Tao Jin, Rangrang Fan, Liangxue Zhou und Gang Guo. „Chitosan for gene delivery: Methods for improvement and applications“. Advances in Colloid and Interface Science 268 (Juni 2019): 25–38. http://dx.doi.org/10.1016/j.cis.2019.03.007.

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45

Ghaemi, Asma, Masoume Vakili-Azghandi, Khalil Abnous, Seyed Mohammad Taghdisi, Mohammad Ramezani und Mona Alibolandi. „Oral non-viral gene delivery platforms for therapeutic applications“. International Journal of Pharmaceutics 642 (Juli 2023): 123198. http://dx.doi.org/10.1016/j.ijpharm.2023.123198.

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Suda, Takeshi, Takeshi Yokoo, Tsutomu Kanefuji, Kenya Kamimura, Guisheng Zhang und Dexi Liu. „Hydrodynamic Delivery: Characteristics, Applications, and Technological Advances“. Pharmaceutics 15, Nr. 4 (31.03.2023): 1111. http://dx.doi.org/10.3390/pharmaceutics15041111.

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The principle of hydrodynamic delivery was initially used to develop a method for the delivery of plasmids into mouse hepatocytes through tail vein injection and has been expanded for use in the delivery of various biologically active materials to cells in various organs in a variety of animal species through systemic or local injection, resulting in significant advances in new applications and technological development. The development of regional hydrodynamic delivery directly supports successful gene delivery in large animals, including humans. This review summarizes the fundamentals of hydrodynamic delivery and the progress that has been made in its application. Recent progress in this field offers tantalizing prospects for the development of a new generation of technologies for broader application of hydrodynamic delivery.

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Houston, Parul, Jo Goodman, Alan Lewis, Callum J. Campbell und Martin Braddock. „Homing markers for atherosclerosis: applications for drug delivery, gene delivery and vascular imaging“. FEBS Letters 492, Nr. 1-2 (07.03.2001): 73–77. http://dx.doi.org/10.1016/s0014-5793(01)02191-3.

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Yui, Nobuhiko. „Supramolecular Approach to Gene Delivery“. Advances in Science and Technology 57 (September 2008): 144–47. http://dx.doi.org/10.4028/www.scientific.net/ast.57.144.

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The most definite feature in polyrotaxanes, in which many cyclic compounds are threaded onto a linear polymeric chains capped with bulky end-groups, is the mobility of cyclic compounds: these cyclic compounds may rotate and/or slide along the polymeric chain. Our previous studies have clarified that the mobility of ligands linked to the cyclic compounds is closely related to enhancing multivalent interaction with biological systems. This concept is now exploiting more practical applications for drug delivery such as gene delivery. We have designed biocleavable polyrotaxanes that have a necklace-like structure between many dimethylaminoethylcarbamoyl-α-cyclodextrins (DMAE-α-CDs) and a disulfide (SS)-introduced poly(ethylene glycol) (PEG) chain. The polyrotaxanes were found to show sufficient cleavage of S-S linkages under reducible condition, which led to triggering pDNA release via the dissociation of the non-covalent linkages between DMAE-α-CDs and the PEG chain. The polyrotaxanes were finally clarified to exhibit great transfection activity as well as non cytotoxicity.

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Wu, Yuanbing, Ania Rashidpour, María Pilar Almajano und Isidoro Metón. „Chitosan-Based Drug Delivery System: Applications in Fish Biotechnology“. Polymers 12, Nr. 5 (21.05.2020): 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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Demirer, Gozde S., Huan Zhang, Natalie S. Goh, Rebecca L. Pinals, Roger Chang und Markita P. Landry. „Carbon nanocarriers deliver siRNA to intact plant cells for efficient gene knockdown“. Science Advances 6, Nr. 26 (Juni 2020): eaaz0495. http://dx.doi.org/10.1126/sciadv.aaz0495.

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Posttranscriptional gene silencing (PTGS) is a powerful tool to understand and control plant metabolic pathways, which is central to plant biotechnology. PTGS is commonly accomplished through delivery of small interfering RNA (siRNA) into cells. Standard plant siRNA delivery methods (Agrobacterium and viruses) involve coding siRNA into DNA vectors and are only tractable for certain plant species. Here, we develop a nanotube-based platform for direct delivery of siRNA and show high silencing efficiency in intact plant cells. We demonstrate that nanotubes successfully deliver siRNA and silence endogenous genes, owing to effective intracellular delivery and nanotube-induced protection of siRNA from nuclease degradation. This study establishes that nanotubes could enable a myriad of plant biotechnology applications that rely on RNA delivery to intact cells.

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