BRC-mediated RNAi targeting of USE1 inhibits tumor growth in vitro and in vivo

Kim, Hye-Jin; Lee, Yeon Kyung; Han, Kyung Ho; Jeon, Hyunsu; Jeong, In-ho; Kim, Sangjin; Lee, Jong Bum; Lee, Peter Chang-Whan

doi:10.1016/j.biomaterials.2019.119630

Cited by 13 publications

(4 citation statements)

References 33 publications

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“…Gene therapy (GT), which uses nucleic acids to treat tumors, is considered a promising therapeutic strategy. − In particular, small-interfering RNAs (siRNAs) have attracted great attention as potential therapeutic agents, because they can regulate protein expression by silencing mRNA (mRNA). , Despite the treatment potential of siRNAs, they have notable limitations, such as fast enzymatic degradation and low intracellular uptake rate in vivo , which necessitate the improvement in delivery to the target. In addition, RNA, owing to its strong negative charge, faces an electrostatic barrier against internalization by cells, which further hinders its therapeutic efficacy in vivo . − Therefore, the combination of GT with other therapeutic methods is considered necessary to compensate for its inherent disadvantages. A promising option for such combination therapy is the use of photothermal therapy (PTT) with GT.…”

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confidence: 99%

Temperature-Sensitive Lipid-Coated Carbon Nanotubes for Synergistic Photothermal Therapy and Gene Therapy

et al. 2021

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The combination of photothermal therapy (PTT) and gene therapy (GT) shows great potential to achieve synergistic anti-tumor activity. However, the lack of a controlled release of genes from carriers remains a severe hindrance. Herein, peptide lipid (PL) and sucrose laurate (SL) were used to coat single-walled carbon nanotubes (SCNTs) and multi-walled carbon nanotubes (MCNTs) to form bifunctional delivery systems (denoted SCNT-PS and MCNT-PS, respectively) with excellent temperature-sensitivity and photothermal performance. CNT/siRNA suppressed tumor growth by silencing survivin expression while exhibiting photothermal effects under near-infrared (NIR) light. SCNT-PS/siRNA showed very high anti-tumor activity, resulting in the complete inhibition of some tumors. It was highly efficient for systemic delivery to tumor sites and to facilitate siRNA release owing to the phase transition of the temperature-sensitive lipids, due to PL and SL coating. Thus, SCNT-PS/siRNA is a promising anti-tumor nanocarrier for combined PTT and GT.

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confidence: 99%

Temperature-Sensitive Lipid-Coated Carbon Nanotubes for Synergistic Photothermal Therapy and Gene Therapy

et al. 2021

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“…75 More recently, bubbled RNA-based cargo (BRC) has been constructed with siRNA targeting UBA6-specific E2 conjugating enzyme 1 (USE1) for lung cancer treatment. 29 The in vivo study showed effectively suppressed transcription of target genes, which leads to tumor growth suppression in A549 tumor xenograft mice treated with BRC.…”

Section: Delivery Of Therapeutics Oligos and Rna Aptamersmentioning

confidence: 98%

“…Copyright 2017 American Chemical Society. Adapted with permission from ref . Copyright 2019 Elsevier Ltd. (C) RNA nanoparticles attached with SEQ with different size (left) and RNA nanoparticles with different shape (right) showed immune stimulation to different extent.…”

Section: Applying Rna Nanotechnology To the Treatment Of Diseasesmentioning

confidence: 99%

Thermostability, Tunability, and Tenacity of RNA as Rubbery Anionic Polymeric Materials in Nanotechnology and Nanomedicine—Specific Cancer Targeting with Undetectable Toxicity

Binzel

Burns

et al. 2021

Chem. Rev.

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RNA nanotechnology is the bottom-up self-assembly of nanometer-scale architectures, resembling LEGOs, composed mainly of RNA. The ideal building material should be (1) versatile and controllable in shape and stoichiometry, (2) spontaneously self-assemble, and (3) thermodynamically, chemically, and enzymatically stable with a long shelf life. RNA building blocks exhibit each of the above. RNA is a polynucleic acid, making it a polymer, and its negative-charge prevents nonspecific binding to negatively charged cell membranes. The thermostability makes it suitable for logic gates, resistive memory, sensor set-ups, and NEM devices. RNA can be designed and manipulated with a level of simplicity of DNA while displaying versatile structure and enzyme activity of proteins. RNA can fold into single-stranded loops or bulges to serve as mounting dovetails for intermolecular or domain interactions without external linking dowels. RNA nanoparticles display rubber- and amoeba-like properties and are stretchable and shrinkable through multiple repeats, leading to enhanced tumor targeting and fast renal excretion to reduce toxicities. It was predicted in 2014 that RNA would be the third milestone in pharmaceutical drug development. The recent approval of several RNA drugs and COVID-19 mRNA vaccines by FDA suggests that this milestone is being realized. Here, we review the unique properties of RNA nanotechnology, summarize its recent advancements, describe its distinct attributes inside or outside the body and discuss potential applications in nanotechnology, medicine, and material science.

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“…Recently, BRC NPs have been successfully applied to target UBA6-specific E2 conjugating enzyme 1 (USE1) and reduce treatment of human lung cancer xenograft in mice upon multiple i.v. injections [ 181 ].…”

Section: Applications Of Rns For Tissue Targetingmentioning

confidence: 99%

RNA nanostructures for targeted drug delivery and imaging

Teodori,

Omer,

Kjems

2024

RNA Biology

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The RNA molecule plays a pivotal role in many biological processes by relaying genetic information, regulating gene expression, and serving as molecular machines and catalyzers. This inherent versatility of RNA has fueled significant advancements in the field of RNA nanotechnology, driving the engineering of complex nanoscale architectures toward biomedical applications, including targeted drug delivery and bioimaging. RNA polymers, serving as building blocks, offer programmability and predictability of Watson-Crick base pairing, as well as non-canonical base pairing, for the construction of nanostructures with high precision and stoichiometry. Leveraging the ease of chemical modifications to protect the RNA from degradation, researchers have developed highly functional and biocompatible RNA architectures and integrated them into preclinical studies for the delivery of payloads and imaging agents. This review offers an educational introduction to the use of RNA as a biopolymer in the design of multifunctional nanostructures applied to targeted delivery in vivo , summarizing physical and biological barriers along with strategies to overcome them. Furthermore, we highlight the most recent progress in the development of both small and larger RNA nanostructures, with a particular focus on imaging reagents and targeted cancer therapeutics in pre-clinical models and provide insights into the prospects of this rapidly evolving field.

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BRC-mediated RNAi targeting of USE1 inhibits tumor growth in vitro and in vivo

Cited by 13 publications

References 33 publications

Temperature-Sensitive Lipid-Coated Carbon Nanotubes for Synergistic Photothermal Therapy and Gene Therapy

Temperature-Sensitive Lipid-Coated Carbon Nanotubes for Synergistic Photothermal Therapy and Gene Therapy

Thermostability, Tunability, and Tenacity of RNA as Rubbery Anionic Polymeric Materials in Nanotechnology and Nanomedicine—Specific Cancer Targeting with Undetectable Toxicity

RNA nanostructures for targeted drug delivery and imaging

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