Nano-Scale Gene Delivery Systems

A special issue of Nanomaterials (ISSN 2079-4991).

Deadline for manuscript submissions: closed (31 December 2020) | Viewed by 28430

Special Issue Editor


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Guest Editor
IBMM, University of Montpellier, CNRS, ENSCM, 34093 Montpellier, France
Interests: photodynamic therapy; two-photon excitation; cancer targeting
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Special Issue Information

Dear Colleagues,

Currently, two big challenges in medicine concern (i) the development of highly efficient and biocompatible nanomaterials and (ii) the use of gene therapy. The coupling of these two fields could lead to an unprecedented technological advance and a therapeutic strategy of rupture, thanks to the development of furtive and targeted nanocargos for gene delivery.

In this Special Issue, we are especially interested in works that describe nanomaterials or polymers that are able to capture nuclei acids and deliver them to the targeted tissue. This delivery could be passive or dependent on an external stimulus. This Special Issue invites manuscripts that provide novel scientific findings on the genetic material delivery of nanomaterials.

Dr. Magali Gary-Bobo
Guest Editor

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Keywords

  • nanomaterials
  • polymers
  • nucleic acids
  • complexation
  • release
  • cell targeting

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Published Papers (4 papers)

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Research

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14 pages, 2290 KiB  
Article
Hydrocarbon-Stapled Peptide Based-Nanoparticles for siRNA Delivery
by Matthieu Simon, Nabila Laroui, Marianne Heyraud, Guillaume Laconde, Lamiaa M. A. Ali, Kevin Bourbiaux, Gilles Subra, Lubomir L. Vezenkov, Baptiste Legrand, Muriel Amblard and Nadir Bettache
Nanomaterials 2020, 10(12), 2334; https://doi.org/10.3390/nano10122334 - 25 Nov 2020
Cited by 5 | Viewed by 3154
Abstract
Small interfering RNAs (siRNAs) are promising molecules for developing new therapies based on gene silencing; however, their delivery into cells remains an issue. In this study, we took advantage of stapled peptide technology that has emerged as a valuable strategy to render natural [...] Read more.
Small interfering RNAs (siRNAs) are promising molecules for developing new therapies based on gene silencing; however, their delivery into cells remains an issue. In this study, we took advantage of stapled peptide technology that has emerged as a valuable strategy to render natural peptides more structured, resistant to protease degradation and more bioavailable, to develop short carriers for siRNA delivery. From the pool of stapled peptides that we have designed and synthesized, we identified non-toxic vectors that were able to efficiently encapsulate siRNA, transport them into the cell and induce gene silencing. Remarkably, the most efficient stapled peptide (JMV6582), is composed of only eight amino-acids and contains only two cationic charges. Full article
(This article belongs to the Special Issue Nano-Scale Gene Delivery Systems)
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11 pages, 2250 KiB  
Article
Biological Assessment of Laser-Synthesized Silicon Nanoparticles Effect in Two-Photon Photodynamic Therapy on Breast Cancer MCF-7 Cells
by Ahmed Al-Kattan, Lamiaa M. A. Ali, Morgane Daurat, Elodie Mattana and Magali Gary-Bobo
Nanomaterials 2020, 10(8), 1462; https://doi.org/10.3390/nano10081462 - 26 Jul 2020
Cited by 14 | Viewed by 2894
Abstract
Driven by their distinctive physiological activities, biological properties and unique theranostic modalities, silicon nanoparticles (SiNPs) are one of the promising materials for the development of novel multifunctional nanoplatforms for biomedical applications. In this work, we assessed the possibility to use laser-synthesized Si NPs [...] Read more.
Driven by their distinctive physiological activities, biological properties and unique theranostic modalities, silicon nanoparticles (SiNPs) are one of the promising materials for the development of novel multifunctional nanoplatforms for biomedical applications. In this work, we assessed the possibility to use laser-synthesized Si NPs as photosensitizers in two-photon excited photodynamic therapy (TPE-PDT) modality. Herein, we used an easy strategy to synthesize ultraclean and monodispersed SiNPs using laser ablation and fragmentation sequences of silicon wafer in aqueous solution, which prevent any specific purification step. Structural analysis revealed the spherical shape of the nanoparticles with a narrow size distribution centered at the mean size diameter of 62 nm ± 0.42 nm, while the negative surface charge of −40 ± 0.3 mV ensured a great stability without sedimentation over a long period of time. In vitro studies on human cancer cell lines (breast and liver) and healthy cells revealed their low cytotoxicity without any light stimulus and their therapeutic potential under TPE-PDT mode at 900 nm with a promising cell death of 45% in case of MCF-7 breast cancer cells, as a consequence of intracellular reactive oxygen species release. Their luminescence emission inside the cells was clearly observed at UV-Vis region. Compared to Si nanoparticles synthesized via chemical routes, which are often linked to additional modules with photochemical and photobiological properties to boost photodynamic effect, laser-synthesized SiNPs exhibit promising intrinsic therapeutic and imaging properties to develop advanced strategy in nanomedicine field. Full article
(This article belongs to the Special Issue Nano-Scale Gene Delivery Systems)
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15 pages, 2163 KiB  
Article
Polythiophenes with Cationic Phosphonium Groups as Vectors for Imaging, siRNA Delivery, and Photodynamic Therapy
by Laure Lichon, Clément Kotras, Bauyrzhan Myrzakhmetov, Philippe Arnoux, Morgane Daurat, Christophe Nguyen, Denis Durand, Karim Bouchmella, Lamiaa Mohamed Ahmed Ali, Jean-Olivier Durand, Sébastien Richeter, Céline Frochot, Magali Gary-Bobo, Mathieu Surin and Sébastien Clément
Nanomaterials 2020, 10(8), 1432; https://doi.org/10.3390/nano10081432 - 22 Jul 2020
Cited by 13 | Viewed by 3722
Abstract
In this work, we exploit the versatile function of cationic phosphonium-conjugated polythiophenes to develop multifunctional platforms for imaging and combined therapy (siRNA delivery and photodynamic therapy). The photophysical properties (absorption, emission and light-induced generation of singlet oxygen) of these cationic polythiophenes were found [...] Read more.
In this work, we exploit the versatile function of cationic phosphonium-conjugated polythiophenes to develop multifunctional platforms for imaging and combined therapy (siRNA delivery and photodynamic therapy). The photophysical properties (absorption, emission and light-induced generation of singlet oxygen) of these cationic polythiophenes were found to be sensitive to molecular weight. Upon light irradiation, low molecular weight cationic polythiophenes were able to light-sensitize surrounding oxygen into reactive oxygen species (ROS) while the highest were not due to its aggregation in aqueous media. These polymers are also fluorescent, allowing one to visualize their intracellular location through confocal microscopy. The most promising polymers were then used as vectors for siRNA delivery. Due to their cationic and amphipathic features, these polymers were found to effectively self-assemble with siRNA targeting the luciferase gene and deliver it in MDA-MB-231 cancer cells expressing luciferase, leading to 30–50% of the gene-silencing effect. In parallel, the photodynamic therapy (PDT) activity of these cationic polymers was restored after siRNA delivery, demonstrating their potential for combined PDT and gene therapy. Full article
(This article belongs to the Special Issue Nano-Scale Gene Delivery Systems)
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Review

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42 pages, 2411 KiB  
Review
Nanomedicines to Deliver mRNA: State of the Art and Future Perspectives
by Itziar Gómez-Aguado, Julen Rodríguez-Castejón, Mónica Vicente-Pascual, Alicia Rodríguez-Gascón, María Ángeles Solinís and Ana del Pozo-Rodríguez
Nanomaterials 2020, 10(2), 364; https://doi.org/10.3390/nano10020364 - 20 Feb 2020
Cited by 150 | Viewed by 18167
Abstract
The use of messenger RNA (mRNA) in gene therapy is increasing in recent years, due to its unique features compared to plasmid DNA: Transient expression, no need to enter into the nucleus and no risk of insertional mutagenesis. Nevertheless, the clinical application of [...] Read more.
The use of messenger RNA (mRNA) in gene therapy is increasing in recent years, due to its unique features compared to plasmid DNA: Transient expression, no need to enter into the nucleus and no risk of insertional mutagenesis. Nevertheless, the clinical application of mRNA as a therapeutic tool is limited by its instability and ability to activate immune responses; hence, mRNA chemical modifications together with the design of suitable vehicles result essential. This manuscript includes a revision of the strategies employed to enhance in vitro transcribed (IVT) mRNA functionality and efficacy, including the optimization of its stability and translational efficiency, as well as the regulation of its immunostimulatory properties. An overview of the nanosystems designed to protect the mRNA and to overcome the intra and extracellular barriers for successful delivery is also included. Finally, the present and future applications of mRNA nanomedicines for immunization against infectious diseases and cancer, protein replacement, gene editing, and regenerative medicine are highlighted. Full article
(This article belongs to the Special Issue Nano-Scale Gene Delivery Systems)
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