2020
DOI: 10.1016/j.addr.2020.07.022
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Recent advances in siRNA delivery mediated by lipid-based nanoparticles

Abstract: Small interfering RNA (siRNA) has been expected to be a unique pharmaceutic for the treatment of broad-spectrum intractable diseases. However, its unfavorable properties such as easy degradation in the blood and negative-charge density are still a formidable barrier for clinical use. For disruption of this barrier, siRNA delivery technology has been significantly advanced in the past two decades. The approval of Patisiran (ONPATTRO™) for the treatment of transthyretin-mediated amyloidosis, the first approved s… Show more

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Cited by 258 publications
(165 citation statements)
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References 210 publications
(230 reference statements)
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“… 1 Oligonucleotides, such as small interfering RNAs (siRNAs), are promising drug modality for a variety of diseases. 2 As a 21–23 bp double-stranded RNA molecule, siRNAs could induce a sequence-specific posttranscriptional gene silencing by targeting complementary nucleotide sequence. 3 However, there are still many obstacles for application of siRNAs in clinical.…”
Section: Introductionmentioning
confidence: 99%
“… 1 Oligonucleotides, such as small interfering RNAs (siRNAs), are promising drug modality for a variety of diseases. 2 As a 21–23 bp double-stranded RNA molecule, siRNAs could induce a sequence-specific posttranscriptional gene silencing by targeting complementary nucleotide sequence. 3 However, there are still many obstacles for application of siRNAs in clinical.…”
Section: Introductionmentioning
confidence: 99%
“…For example, these naked tools are easily degraded and exhibit low delivery in the cellular environment. However, some investigations suggest that the conjugation of these tools with NSs could improve their therapeutic action [ 94 , 95 , 154 , 159 , 160 , 161 , 162 ].…”
Section: Nanotechnology Approaches For Hiv/aids Treatmentmentioning
confidence: 99%
“…The small interfering RNAs (siRNAs), are a promising approach for gene therapy, however their intracellular administration remains difficult. To date, a great variety of delivery system for siRNAs has been investigated such as lipids, polymers, aptamers and cell penetrating peptides (CPPs) [ 26 , 27 , 28 , 29 , 30 , 31 , 32 , 33 , 34 , 35 , 36 , 37 , 38 , 39 , 40 , 41 , 42 , 43 , 44 ]. In this regard, CPPs appear as a new class of transporters, which increase intracellular uptake of biologically active molecules including siRNAs [ 45 ] ( Figure 2 ).…”
Section: Benefit Of Cpps Used As Vehicles In Oligonucleotides Delimentioning
confidence: 99%