Microbubbles for Nucleic Acid Delivery in Liver Using Mild Sonoporation

Mignet, Nathalie; Marie, Corinne; Delalande, Anthony; Manta, Simona; Bureau, Michel-Francis; Renault, Gilles; Scherman, Daniel; Pichon, Chantal

doi:10.1007/978-1-4939-9092-4_25

Cited by 8 publications

(6 citation statements)

References 19 publications

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“…Physical/mechanical methods, such as electroporation [13][14][15], sonoporation [16][17][18], magnetofection [19,20], optoporation [21,22], gene gun [23], and microinjection [24][25][26], attempt to force naked NAs into the cytosol or nucleus to achieve successful transfection [27,28]. Although conceptually simple yet powerful means for transfecting cells, such methods are however expensive and somehow inconvenient for most gene delivery applications [29].…”

Section: Nucleic Acid Description Site Of Action Applications/pathwaymentioning

confidence: 99%

Non-Viral in Vitro Gene Delivery: It is Now Time to Set the Bar!

et al. 2020

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Transfection by means of non-viral gene delivery vectors is the cornerstone of modern gene delivery. Despite the resources poured into the development of ever more effective transfectants, improvement is still slow and limited. Of note, the performance of any gene delivery vector in vitro is strictly dependent on several experimental conditions specific to each laboratory. The lack of standard tests has thus largely contributed to the flood of inconsistent data underpinning the reproducibility crisis. A way researchers seek to address this issue is by gauging the effectiveness of newly synthesized gene delivery vectors with respect to benchmarks of seemingly well-known behavior. However, the performance of such reference molecules is also affected by the testing conditions. This survey points to non-standardized transfection settings and limited information on variables deemed relevant in this context as the major cause of such misalignments. This review provides a catalog of conditions optimized for the gold standard and internal reference, 25 kDa polyethyleneimine, that can be profitably replicated across studies for the sake of comparison. Overall, we wish to pave the way for the implementation of standardized protocols in order to make the evaluation of the effectiveness of transfectants as unbiased as possible.

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Section: Nucleic Acid Description Site Of Action Applications/pathwaymentioning

confidence: 99%

Non-Viral in Vitro Gene Delivery: It is Now Time to Set the Bar!

et al. 2020

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“…Sonoporation has been well-regarded as an emerging membrane perforation paradigm in cancer therapeutics 1,2 and drug/gene delivery [3][4][5] . This membrane perforation paradigm often works by applying low-intensity ultrasound to microbubble situated near the cell membrane to create transient pores that in turn serve as physical windows for extracellular molecules to infiltrate target cells without inducing undesired thermal effects 6 .…”

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confidence: 99%

Sonoporation generates downstream cellular impact after membrane resealing

Duan

Zhou

Wan

et al. 2021

Sci Rep

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Sonoporation via microbubble-mediated ultrasound exposure has shown potential in drug and gene delivery. However, there is a general lack of mechanistic knowledge on sonoporation-induced cellular impact after membrane resealing, and this issue has made it challenging to apply sonoporation efficiently in practice. Here, we present new evidence on how sonoporation, without endangering immediate cell viability, may disrupt downstream cellular hemostasis in ways that are distinguished from the bioeffects observed in other sonicated and unsonoporated cells. Sonoporation was realized on HL-60 leukemia cells by delivering pulsed ultrasound (1 MHz frequency, 0.50 MPa peak negative pressure; 10% duty cycle; 30 s exposure period; 29.1 J/cm2 acoustic energy density) in the presence of lipid-shelled microbubbles (1:1 cell-to-bubble ratio). Results showed that 54.6% of sonoporated cells, despite remaining initially viable, underwent apoptosis or necrosis at 24 h after sonoporation. Anti-proliferation behavior was also observed in sonoporated cells as their subpopulation size was reduced by 43.8% over 24 h. Preceding these cytotoxic events, the percentages of sonoporated cells in different cell cycle phases were found to be altered by 12 h after exposure. As well, for sonoporated cells, their expressions of cytoprotective genes in the heat shock protein-70 (HSP-70) family were upregulated by at least 4.1 fold at 3 h after exposure. Taken altogether, these findings indicate that sonoporated cells attempted to restore homeostasis after membrane resealing, but many of them ultimately failed to recover. Such mechanistic knowledge should be taken into account to devise more efficient sonoporation-mediated therapeutic protocols.

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“…2) Sonoporation: While the data for using this technique is limited for CRISPR technologies, still a study by Yoon et al utilized very high frequency ultrasound near cell membranes to deliver CRISPR/Cas payloads into cells and nucleus with precision [70]. In general this acoustic transfection mechanism of gene transfer modality has been used in many other ways for gene therapy [71,72].…”

Section: A) Physical Methodsmentioning

confidence: 99%

Recent Advancement and Innovations in CRISPR/Cas and CRISPR Related Technologies: A review

Khan¹

2021

BTBP

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CRISPR genome editing technologies have been improving by every passing day. The initial CRISPR/Cas9 technologies, though emerged an improved version of genome editing in competition with TALENS and ZFNs, was nevertheless not free from technical and off-target effects. Technological improvements overtime start addressing issues with original CRISPR/Cas9 technology. The major areas of improvement targeted nucleases and delivery methods. Overtime the nuclease like Cas9 had some modifications like FokI-dCas9, Truncated guide RNAs (tru-gRNAs), Paired Cas9 nickase, Cpf1, Cas6 with Csm/Csr complex and chemically treated Cas9. In terms of delivery methods the improvements came along after almost all methods including viral methods like Recombinant Adeno Associated Viruses (rAAV), Lentivirus (LV), and bacteriophages. The review summarizes various non-viral gene delivery modes including physical methods like electroporation and chemical methods like nano particles, cell-derived membrane vesicles (CMVs) with upgraded developments. The review also compares various modes of delivering CRISPR gene editing machinery.

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Microbubbles for Nucleic Acid Delivery in Liver Using Mild Sonoporation

Cited by 8 publications

References 19 publications

Non-Viral in Vitro Gene Delivery: It is Now Time to Set the Bar!

Non-Viral in Vitro Gene Delivery: It is Now Time to Set the Bar!

Sonoporation generates downstream cellular impact after membrane resealing

Recent Advancement and Innovations in CRISPR/Cas and CRISPR Related Technologies: A review

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