In Vitro CRISPR/Cas9 Transfection and Gene-Editing Mediated by Multivalent Cationic Liposome–DNA Complexes

Sousa, D. Z.; Gaspar, Ricardo; Ferreira, Celso J. O.; Baltazar, Fátima; Rodrigues, L. R.; Silva, Bruno F. B.

doi:10.3390/pharmaceutics14051087

Cited by 16 publications

(9 citation statements)

References 70 publications

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“…When permanent cationic lipid-DOTAP is applied, the poor transfection efficiency of dLNP is likely attributed to the inefficient intracellular release of encapsulated siRNA into the cytoplasm. This finding is in agreement with another study reporting the poor transfection efficiency of DOTAP-containing lipoplexes, which corroborated to inefficient DNA release from DOTAP cationic vesicles into the cytoplasm [ 43 ]. Yung et al reported a combination formulation with amine and tertiary amine cationic lipids, which achieved superior delivery of microRNA-21 [ 44 ].…”

Section: Discussionsupporting

confidence: 93%

Development of a Combined Lipid-Based Nanoparticle Formulation for Enhanced siRNA Delivery to Vascular Endothelial Cells

Plantinga

et al. 2022

Pharmaceutics

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Low transfection efficiency in endothelial cells (EC) is still a bottleneck for the majority of siRNA-based vascular delivery approaches. In this work, we developed a lipid-based nanoparticle (LNP) formulation based on a combination of a permanently charged cationic lipid-DOTAP and a conditionally ionized cationic lipid-MC3 (DOTAP/MC3) for the enhanced delivery of siRNA into EC. Compared with a single DOTAP or MC3-based benchmark LNP, we demonstrated that the DOTAP/MC3 LNP formulation shows the best transfection efficiency both in primary EC in vitro and in endothelium in zebrafish. The high transfection activity of the DOTAP/MC3 LNP formulation is achieved by a combination of improved endothelial association mediated by DOTAP and MC3-triggered efficient siRNA intracellular release in EC. Furthermore, AbVCAM-1-coupled DOTAP/MC3 LNP-mediated siRNARelA transfection showed pronounced anti-inflammatory effects in inflammatory-activated primary EC by effectively blocking the NF-κB pathway. In conclusion, the combination of permanent and ionizable cationic lipids in LNP formulation provides an effective endothelial cell delivery of siRNA.

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Section: Discussionsupporting

confidence: 93%

Development of a Combined Lipid-Based Nanoparticle Formulation for Enhanced siRNA Delivery to Vascular Endothelial Cells

Plantinga

et al. 2022

Pharmaceutics

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“…These vesicles excel in transmitting genetic material into target cells, facilitating the delivery of nucleic acids such as DNA or RNA [ 98 , 102 ]. The efficiency of this process, known as transfection, is vital in manipulating cellular functions and studying gene expression [ 104 ]. Liposomes, with their ability to encapsulate and protect genetic material, enhance transfection efficiency by ensuring the successful integration of foreign genetic material into the host cells.…”

Section: Discussionmentioning

confidence: 99%

Liposome-Based Drug Delivery Systems in Cancer Research: An Analysis of Global Landscape Efforts and Achievements

Hamad,

Harb,

Bustanji

2024

Pharmaceutics

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Lipid-bilayer-based liposomes are gaining attention in scientific research for their versatile role in drug delivery. With their amphiphilic design, liposomes efficiently encapsulate and deliver drugs to targeted sites, offering controlled release. These artificial structures hold great promise in advancing cancer therapy methodologies. Bibliometric research analyzes systematic literary data statistically. This study used bibliometric indicators to examine, map, and evaluate the applications of liposomes in cancer therapy. A Scopus search was conducted to identify all English-language peer-reviewed scientific publications on the applications of liposomes in cancer therapy within the past twenty years. Bibliometric indicators were calculated using VOSviewer and Biblioshiny. We produced thematic, conceptual, and visualization charts. A total of 14,873 published documents were obtained. The procedure of keyword mapping has effectively identified the main areas of research concentration and prevailing trends within this specific field of study. The significant clusters discovered through theme and hotspot analyses encompassed many topics such as the use of multiple strategies in chemotherapy and different forms of cancer, the study of pharmacokinetics and nanomedicine, as well as the investigation of targeted drug delivery, cytotoxicity, and gene delivery. Liposomes were employed as drug delivery systems so as to selectively target cancer cells and improve the bioavailability of anticancer drugs. The work showcased the capacity to tailor these liposomes for accurate drug delivery by including potent anticancer medications. Our findings not only bring attention to the latest progress in utilizing liposomes for cancer treatment but also underscore the vital need for ongoing research, collaborative efforts, and the effective translation of these breakthroughs into tangible clinical applications, emphasizing the dynamic and evolving nature of cancer therapeutics.

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“…Despite early challenges of low transfection efficiency, liposome formulations have been developed to deliver siRNA, plasmid DNA, and mRNA for gene silencing, replacement, and protein expression [ 25 , 26 , 27 , 28 , 29 , 30 , 31 , 32 , 33 , 34 ]. Surface modifications of liposomes with ligands, such as antibodies or peptides, have been a focus of research, enabling selective binding to specific receptors on target cells and improving delivery efficiency [ 12 , 13 , 35 , 36 , 37 , 38 ]. Additionally, stimuli-responsive liposomes were engineered to release their cargo in response to environmental cues, such as pH changes or ultrasound activity [ 37 , 39 , 40 , 41 ].…”

Section: Liposome-based Delivery Systemmentioning

confidence: 99%

“…Liposomes have been extensively engineered to optimize the delivery of CRISPR/Cas and gRNA [ 12 , 13 , 35 , 36 , 37 , 40 , 50 , 51 , 52 ]. The combination of the CRISPR-based system with liposome delivery technology enables precise and efficient genetic modifications in cells and tissues.…”

Section: Liposome-based Crispr Deliverymentioning

confidence: 99%

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Liposome-Based Carriers for CRISPR Genome Editing

Yin

Harmancey

McPherson

et al. 2023

IJMS

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The CRISPR-based genome editing technology, known as clustered regularly interspaced short palindromic repeats (CRISPR), has sparked renewed interest in gene therapy. This interest is accompanied by the development of single-guide RNAs (sgRNAs), which enable the introduction of desired genetic modifications at the targeted site when used alongside the CRISPR components. However, the efficient delivery of CRISPR/Cas remains a challenge. Successful gene editing relies on the development of a delivery strategy that can effectively deliver the CRISPR cargo to the target site. To overcome this obstacle, researchers have extensively explored non-viral, viral, and physical methods for targeted delivery of CRISPR/Cas9 and a guide RNA (gRNA) into cells and tissues. Among those methods, liposomes offer a promising approach to enhance the delivery of CRISPR/Cas and gRNA. Liposomes facilitate endosomal escape and leverage various stimuli such as light, pH, ultrasound, and environmental cues to provide both spatial and temporal control of cargo release. Thus, the combination of the CRISPR-based system with liposome delivery technology enables precise and efficient genetic modifications in cells and tissues. This approach has numerous applications in basic research, biotechnology, and therapeutic interventions. For instance, it can be employed to correct genetic mutations associated with inherited diseases and other disorders or to modify immune cells to enhance their disease-fighting capabilities. In summary, liposome-based CRISPR genome editing provides a valuable tool for achieving precise and efficient genetic modifications. This review discusses future directions and opportunities to further advance this rapidly evolving field.

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In Vitro CRISPR/Cas9 Transfection and Gene-Editing Mediated by Multivalent Cationic Liposome–DNA Complexes

Cited by 16 publications

References 70 publications

Development of a Combined Lipid-Based Nanoparticle Formulation for Enhanced siRNA Delivery to Vascular Endothelial Cells

Development of a Combined Lipid-Based Nanoparticle Formulation for Enhanced siRNA Delivery to Vascular Endothelial Cells

Liposome-Based Drug Delivery Systems in Cancer Research: An Analysis of Global Landscape Efforts and Achievements

Liposome-Based Carriers for CRISPR Genome Editing

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