Biological recognition and cellular trafficking of targeted RNA-lipid nanoparticles

Escalona-Rayo, Oscar; Papadopoulou, Panagiota; Slütter, Bram; Kros, Alexander

doi:10.1016/j.copbio.2023.103041

Cited by 4 publications

(2 citation statements)

References 57 publications

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“…A delivery system with optimal efficacy in in vivo genome editing is based on the use of recombinant adeno-associated virus (AAV), but may cause adverse effects ( Dubey and Mostafavi, 2023 ). Therefore, lipid NPs represent an alternative ( Escalona-Rayo et al, 2023 ). For example, lipid NPs have been used to target macrophages in mice using the CRISPR/Cas9 system, although only a limited percentage (20%) of genome editing was observed ( Escalona-Rayo et al, 2023 ).…”

Section: Crispr/cas9 Editing and Base Primersmentioning

confidence: 99%

“…Therefore, lipid NPs represent an alternative ( Escalona-Rayo et al, 2023 ). For example, lipid NPs have been used to target macrophages in mice using the CRISPR/Cas9 system, although only a limited percentage (20%) of genome editing was observed ( Escalona-Rayo et al, 2023 ). Therefore, it is a challenge to induce robust genome editing in target tissues, such as the aorta.…”

Section: Crispr/cas9 Editing and Base Primersmentioning

confidence: 99%

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The key role of miRNA in syndromic and sporadic forms of ascending aortic aneurysms as biomarkers and targets of novel therapeutic strategies

Terriaca,

Monastero,

Orlandi

et al. 2024

Front. Genet.

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Increasing evidence shows that epigenetics also plays a key role in regulating the pathogenetic mechanism of all types of aortic aneurysms. It is well-known that epigenetic factors modulate gene expression. This mechanism appears to be of interest especially knowing the relevance of genetic susceptibility and genetic factors in the complex pathophysiology of aortic aneurysms, and of sporadic forms; in fact, the latter are the result of a close interaction between genetic and modifiable lifestyle factors (i.e., nutrition, smoking, infections, use of drugs, alcohol, sedentary lifestyle, etc.). Epigenetic factors include DNA methylation, post-translational histone modifications, and non-coding RNA. Here, our attention is focused on the role of miRNA in syndromic and sporadic forms of thoracic aortic aneurysms. They could be both biomarkers and targets of novel therapeutic strategies.

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Section: Crispr/cas9 Editing and Base Primersmentioning

confidence: 99%

Section: Crispr/cas9 Editing and Base Primersmentioning

confidence: 99%

The key role of miRNA in syndromic and sporadic forms of ascending aortic aneurysms as biomarkers and targets of novel therapeutic strategies

Terriaca,

Monastero,

Orlandi

et al. 2024

Front. Genet.

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In Vivo Endothelial Cell Gene Silencing by siRNA‐LNPs Tuned with Lipoamino Bundle Chemical and Ligand Targeting

Yazdi,

Pöhmerer,

Hasanzadeh Kafshgari

et al. 2024

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Although small‐interfering RNAs (siRNAs) are specific silencers for numerous disease‐related genes, their clinical applications still require safe and effective means of delivery into target cells. Highly efficient lipid nanoparticles (LNPs) are developed for siRNA delivery, showcasing the advantages of novel pH‐responsive lipoamino xenopeptide (XP) carriers. These sequence‐defined XPs are assembled by branched lysine linkages between cationizable polar succinoyl tetraethylene pentamine (Stp) units and apolar lipoamino fatty acids (LAFs) at various ratios into bundle or U‐shape topologies. Formulation of siRNA‐LNPs using LAF4‐Stp1 XPs as ionizable compounds led to robust cellular uptake, high endosomal escape, and successful in vitro gene silencing activity at an extremely low (150 picogram) siRNA dose. Of significance is the functional in vivo endothelium tropism of siRNA‐LNPs with bundle LAF4‐Stp1 XP after intravenous injection into mice, demonstrated by superior knockdown of liver sinusoidal endothelial cell (LSEC)‐derived factor VIII (FVIII) and moderate silencing of hepatocyte‐derived FVII compared to DLin‐MC3‐DMA‐based LNPs. Optimizing lipid composition following click‐modification of siRNA‐LNPs with ligand c(RGDfK) efficiently silenced vascular endothelial growth factor receptor‐2 (VEGFR‐2) in tumor endothelial cells (TECs). The findings shed light on the role of ionizable XPs in the LNP in vivo cell‐type functional targeting, laying the groundwork for future therapeutic applications.

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RNA-based logic for selective protein expression in senescent cells

Jacobs,

Khalifeh,

Koot

et al. 2024

The International Journal of Biochemistry & Cell Biology

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Biological recognition and cellular trafficking of targeted RNA-lipid nanoparticles

Cited by 4 publications

References 57 publications

The key role of miRNA in syndromic and sporadic forms of ascending aortic aneurysms as biomarkers and targets of novel therapeutic strategies

The key role of miRNA in syndromic and sporadic forms of ascending aortic aneurysms as biomarkers and targets of novel therapeutic strategies

In Vivo Endothelial Cell Gene Silencing by siRNA‐LNPs Tuned with Lipoamino Bundle Chemical and Ligand Targeting

RNA-based logic for selective protein expression in senescent cells

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